부산대학교 도서관

Choudhury, Sourav R ; Fitzpatrick, Zachary ; Harris, Anne F ; Maitland, Stacy A ; Ferreira, Jennifer S ; Zhang, Yuanfan ; Ma, Shan ; Sharma, Rohit B ; Gray-Edwards, Heather L ; Johnson, Jacob A ; Johnson, Aime K ; Alonso, Laura C ; Punzo, Claudio ; Wagner, Kathryn R ; Maguire, Casey A ; Kotin, Robert M ; Martin, Douglas R ; Sena-Esteves, Miguel

In Molecular Therapy July 2016 24(7):1247-1257

Crommentuijn, Matheus HW ; Kantar, Rami ; Noske, David P ; Vandertop, W Peter ; Badr, Christian E ; Würdinger, Thomas ; Maguire, Casey A ; Tannous, Bakhos A

In Molecular Therapy - Oncolytics 2016 3

Dashkoff, Jonathan ; Lerner, Eli P ; Truong, Nhi ; Klickstein, Jacob A ; Fan, Zhanyun ; Mu, Dakai ; Maguire, Casey A ; Hyman, Bradley T ; Hudry, Eloise

In Molecular Therapy - Methods & Clinical Development 2016 3

Gong, Yi ; Mu, Dakai ; Prabhakar, Shilpa ; Moser, Ann ; Musolino, Patricia ; Ren, JiaQian ; Breakefield, Xandra O ; Maguire, Casey A ; Eichler, Florian S

In Molecular Therapy May 2015 23(5):824-834

Chen, Walter W ; Balaj, Leonora ; Liau, Linda M ; Samuels, Michael L ; Kotsopoulos, Steve K ; Maguire, Casey A ; LoGuidice, Lori ; Soto, Horacio ; Garrett, Matthew ; Zhu, Lin Dan ; Sivaraman, Sarada ; Chen, Clark ; Wong, Eric T ; Carter, Bob S ; Hochberg, Fred H ; Breakefield, Xandra O ; Skog, Johan

In Molecular Therapy - Nucleic Acids 2013 2

Maguire, Casey A ; Bovenberg, M Sarah ; Crommentuijn, Matheus HW ; Niers, Johanna M ; Kerami, Mariam ; Teng, Jian ; Sena-Esteves, Miguel ; Badr, Christian E ; Tannous, Bakhos A

In Molecular Therapy - Nucleic Acids 2013 2

Maguire, Casey A ; Meijer, Dimphna H ; LeRoy, Stanley G ; Tierney, Laryssa A ; Broekman, Marike LD ; Costa, Fabricio F ; Breakefield, Xandra O ; Stemmer-Rachamimov, Anat ; Sena-Esteves, Miguel

In Molecular Therapy October 2008 16(10):1695-1702

Henriques C; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal.; Albuquerque P; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal.; Rufino-Ramos D; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal; Center for Genomic Medicine and Department of Pathology, Massachusetts General Hospital, Boston, MA 02115, USA; Department of Pathology, Massachusetts General Hospital, Boston, MA 02114, USA; Department of Pathology, Harvard Medical School, Boston, MA 02115, USA.; Lopes MM; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.; Leandro K; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal.; Miranda CO; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.; Almeida R; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal.; Lopes-Gabriel G; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal.; de Sousa-Lourenço J; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal.; Lopes SM; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.; Gaspar LS; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.; Lobo DD; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.; Silva AC; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.; Ribeiro-Rodrigues TM; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Coimbra Institute for Clinical and Biomedical Research (iCBR), University of Coimbra, 3004-504 Coimbra, Portugal; Clinical Academic Center of Coimbra (CACC), Coimbra, Portugal; Faculty of Medicine, University of Coimbra, 3000-548 Coimbra, Portugal.; Girão H; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Coimbra Institute for Clinical and Biomedical Research (iCBR), University of Coimbra, 3004-504 Coimbra, Portugal; Clinical Academic Center of Coimbra (CACC), Coimbra, Portugal; Faculty of Medicine, University of Coimbra, 3000-548 Coimbra, Portugal.; Gomes CM; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; Coimbra Institute for Clinical and Biomedical Research (iCBR), University of Coimbra, 3004-504 Coimbra, Portugal; Clinical Academic Center of Coimbra (CACC), Coimbra, Portugal; Faculty of Medicine, University of Coimbra, 3000-548 Coimbra, Portugal.; Baganha R; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.; Duarte S; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.; Maguire CA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA 02129, USA.; Santana MM; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal.; Pereira de Almeida L; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Faculty of Pharmacy, University of Coimbra, 3000-548 Coimbra, Portugal. Electronic address: luispa@cnc.uc.pt.; Nobre RJ; Gene and Stem Cell Therapies for the Brain Group, Center for Neuroscience and Cell Biology, University of Coimbra (CNC-UC), 3004-504 Coimbra, Portugal; Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, 3004-504 Coimbra, Portugal; GeneT, Center for Excellence in Gene Therapy in Portugal, University of Coimbra, 3004-504 Coimbra, Portugal; ViraVector-Viral Vectors for Gene Transfer Core Facility, University of Coimbra, 3004-504 Coimbra, Portugal; Institute for Interdisciplinary Research (iiiUC), University of Coimbra, 3030-789 Coimbra, Portugal. Electronic address: rui.nobre@cnc.uc.pt.

Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE

Alves CRR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Das S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Krishnan V; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Ha LL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Fox LR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Stutzman HE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Shamber CE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Kalailingam P; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; McCarthy S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Lino Cardenas CL; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Fong CE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Imai T; Neurovascular Research Unit, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA.; Mitra S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Yun S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Wood RK; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.; Benning FMC; Department of Molecular Biology, Massachusetts General Hospital, Boston, MA, USA.; Department of Genetics, Harvard Medical School, Boston, MA, USA.; Roh K; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Lawton J; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Kim N; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; Silverstein RA; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Biological and Biomedical Sciences Program, Harvard University, Boston, MA, USA.; Ferreira da Silva J; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; de la Cruz D; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Richa R; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Xie J; Department of Genetics and Cellular Medicine, UMass Chan Medical School, Worcester, MA, USA.; Gray-Edwards HL; Department of Genetics and Cellular Medicine, UMass Chan Medical School, Worcester, MA, USA.; Malhotra R; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Chung DY; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Neurovascular Research Unit, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Chao LH; Department of Molecular Biology, Massachusetts General Hospital, Boston, MA, USA.; Department of Genetics, Harvard Medical School, Boston, MA, USA.; Tsai SQ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.; Maguire CA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Lindsay ME; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. Lindsay.Mark@mgh.harvard.edu.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. Lindsay.Mark@mgh.harvard.edu.; Kleinstiver BP; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.; Department of Pathology, Harvard Medical School, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.; Musolino PL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA. pmusolino@mgh.harvard.edu.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA. pmusolino@mgh.harvard.edu.; Department of Neurology, Harvard Medical School, Boston, MA, USA. pmusolino@mgh.harvard.edu.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA. pmusolino@mgh.harvard.edu.

Publisher: Springer Nature Country of Publication: England NLM ID: 101696896 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2157-846X (Electronic) Linking ISSN: 2157846X NLM ISO Abbreviation: Nat Biomed Eng Subsets: MEDLINE

Abou Haidar E; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; Prabhakar S; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; Cheah PS; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; Department of Human Anatomy, Faculty of Medicine and Health Sciences, Universiti Putra Malaysia, Serdang, Malaysia.; Hanlon KS; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; Department of Neurobiology, Harvard Medical School, Boston, MA, USA.; EGA-Institute for Women's Health, University College London, London, UK.; Transduction Consulting, London, UK.; Espinoza P; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; Crain AV; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; Patel N; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; Radcliff GW; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; Cheng M; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; Hernández IC; Surgical Photonics & Engineering Laboratory, Mass Eye and Ear, Harvard Medical School, Boston, MA, 02114, USA.; Minderler S; Surgical Photonics & Engineering Laboratory, Mass Eye and Ear, Harvard Medical School, Boston, MA, 02114, USA.; de la Cruz D; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; Ng C; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; da Hora CC; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.; Mayo Clinic College of Medicine and Science, Jacksonville, FL, 32224, USA.; Charest A; Department of Medicine, Harvard Medical School, Boston, MA, 02215, USA.; Cancer Research Institute, Beth Israel Deaconess Medical Center, Boston, MA, 02215, USA.; Stemmer-Rachamimov A; Department of Pathology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Jowett N; Surgical Photonics & Engineering Laboratory, Mass Eye and Ear, Harvard Medical School, Boston, MA, 02114, USA.; Breakefield XO; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA. breakefield@hms.harvard.edu.; Maguire CA; Molecular Neurogenetics Unit, Department of Neurology, The Massachusetts General Hospital, Charlestown MA and Program in Neuroscience, Harvard Medical School, Boston, MA, USA. cmaguire@mgh.harvard.edu.

Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE

Gong Y; Massachusetts General Hospital, Department of Neurology, Harvard Medical School, Boston, MA, USA.; Laheji F; Massachusetts General Hospital, Department of Neurology, Harvard Medical School, Boston, MA, USA.; Berenson A; Massachusetts General Hospital, Department of Neurology, Harvard Medical School, Boston, MA, USA.; Li Y; Massachusetts General Hospital, Department of Neurology, Harvard Medical School, Boston, MA, USA.; Moser A; Peroxisome Disease Lab, Hugo W Moser Research Institute, Baltimore, MD, USA.; Qian A; Massachusetts General Hospital, Department of Neurology, Harvard Medical School, Boston, MA, USA.; Frosch M; Massachusetts General Hospital, Department of Neuropathology, Harvard Medical School, Boston, MA, USA.; Sadjadi R; Massachusetts General Hospital, Department of Neurology, Harvard Medical School, Boston, MA, USA.; Hahn R; Massachusetts General Hospital, Department of Neurology, Harvard Medical School, Boston, MA, USA.; Maguire CA; Massachusetts General Hospital, Department of Neurology, Harvard Medical School, Boston, MA, USA.; Eichler F; Massachusetts General Hospital, Department of Neurology, Harvard Medical School, Boston, MA, USA.

Publisher: Wiley-Liss Country of Publication: United States NLM ID: 7707449 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1531-8249 (Electronic) Linking ISSN: 03645134 NLM ISO Abbreviation: Ann Neurol Subsets: MEDLINE

Maguire CA; Department of Neurology, The Massachusetts General Hospital, Charlestown, MA, USA.; Harvard Medical School, Boston, MA, USA.

Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE

Griciuc, Ana; Federico, Anthony N; Natasan, Jeyashree; Forte, Angela M; McGinty, Danielle; Nguyen, Huong; Volak, Adrienn; LeRoy, Stanley; Gandhi, Sheetal; Lerner, Eli P; Hudry, Eloise; Tanzi, Rudolph E; Maguire, Casey A

Human Molecular Genetics; Sep2020, Vol. 29 Issue 17, p2920-2935, 16p

Meliani, Amine ; Fitzpatrick, Zachary ; Boisgerault, Florence ; Ronzitti, Giuseppe ; Collaud, Fanny ; Charles, Severine ; Maguire, Casey A ; Mingozzi, Federico

In Molecular Therapy May 2016 24 Supplement 1:S235-S236

Espinoza P; Harvard Medical School, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA, USA.; Cheng M; Harvard Medical School, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA, USA.; Ng C; Harvard Medical School, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA, USA.; Cruz D; Harvard Medical School, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA, USA.; Wasson ED; FSU Institute for Pediatric Rare Diseases, College of Medicine, Florida State University, Tallahassee, FL, USA.; McCarthy DM; FSU Institute for Pediatric Rare Diseases, College of Medicine, Florida State University, Tallahassee, FL, USA.; Bhide PG; FSU Institute for Pediatric Rare Diseases, College of Medicine, Florida State University, Tallahassee, FL, USA.; Maguire CA; Harvard Medical School, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA, USA. Electronic address: cmaguire@mgh.harvard.edu.; Santoscoy MC; Harvard Medical School, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA, USA. Electronic address: miguel.santoscoy@merck.com.

Publisher: Academic Press Country of Publication: Belgium NLM ID: 9815657 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1096-7184 (Electronic) Linking ISSN: 10967176 NLM ISO Abbreviation: Metab Eng Subsets: MEDLINE

Cheng M; Department of Neurology, Massachusetts General Hospital, Boston, MA 02114, USA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA.; Harvard Medical School, Boston, MA 02115, USA.; Cruz D; Department of Neurology, Massachusetts General Hospital, Boston, MA 02114, USA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA.; Harvard Medical School, Boston, MA 02115, USA.; Crain AV; Department of Neurology, Massachusetts General Hospital, Boston, MA 02114, USA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA.; Harvard Medical School, Boston, MA 02115, USA.; Espinoza P; Department of Neurology, Massachusetts General Hospital, Boston, MA 02114, USA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA.; Harvard Medical School, Boston, MA 02115, USA.; Ng C; Department of Neurology, Massachusetts General Hospital, Boston, MA 02114, USA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA.; Harvard Medical School, Boston, MA 02115, USA.; Elmore ZC; Department of Surgery, Duke University School of Medicine, Durham, NC 27710, USA.; Asokan A; Department of Surgery, Duke University School of Medicine, Durham, NC 27710, USA.; Department of Molecular Genetics & Microbiology, Duke University School of Medicine, Durham, NC 27710, USA.; Department of Biomedical Engineering, Duke University, Durham, NC 27710, USA.; Maguire CA; Department of Neurology, Massachusetts General Hospital, Boston, MA 02114, USA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA.; Harvard Medical School, Boston, MA 02115, USA.

Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE

Nieland L; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02129, USA; Department of Cell and Chemical Biology, Leiden University Medical Center, 2300 RC Leiden, the Netherlands.; Vrijmoet AB; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02129, USA.; Jetten IW; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02129, USA.; Rufino-Ramos D; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA; Department of Pathology, Massachusetts General Hospital, Boston, MA 02114, USA; Department of Pathology, Harvard Medical School, Boston, MA 02115, USA.; de Reus AJEM; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02129, USA.; Breyne K; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02129, USA.; Kleinstiver BP; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA; Department of Pathology, Massachusetts General Hospital, Boston, MA 02114, USA; Department of Pathology, Harvard Medical School, Boston, MA 02115, USA.; Maguire CA; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02129, USA; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Harvard Medical School, Boston, MA 02116, USA.; Broekman MLD; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02129, USA; Department of Cell and Chemical Biology, Leiden University Medical Center, 2300 RC Leiden, the Netherlands; Department of Neurosurgery, Haaglanden Medical Center, 2512 VA The Hague, the Netherlands; Department of Neurosurgery, Leiden University Medical Center, 2300 RC Leiden, the Netherlands.; Breakefield XO; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02129, USA.; Abels ER; Department of Cell and Chemical Biology, Leiden University Medical Center, 2300 RC Leiden, the Netherlands. Electronic address: e.r.abels@lumc.nl.

Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE

Ahmed, Sherif G; Abdelnabi, Ahmed; Maguire, Casey A; Doha, Mohamed; Sagers, Jessica E; Lewis, Rebecca M; Muzikansky, Alona; Giovannini, Marco; Stemmer-Rachamimov, Anat; Stankovic, Konstantina M; Fulci, Giulia; Brenner, Gary J

Neuro-Oncology; Jul2019, Vol. 21 Issue 7, p854-866, 13p

Alves CRR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Das S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Krishnan V; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Ha LL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Fox LR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Stutzman HE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Shamber CE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Kalailingam P; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; McCarthy S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Lino Cardenas CL; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Fong CE; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Imai T; Neurovascular Research Unit, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA.; Mitra S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Yun S; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Wood RK; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.; Benning FMC; Department of Molecular Biology, Massachusetts General Hospital, Boston, MA, USA.; Department of Genetics, Harvard Medical School, Boston, MA USA.; Lawton J; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Kim N; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; Silverstein RA; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Biological and Biomedical Sciences Program, Harvard, Boston, MA, USA.; da Silva JF; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; de la Cruz D; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Richa R; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Malhotra R; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Chung DY; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Neurovascular Research Unit, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Chao LH; Department of Molecular Biology, Massachusetts General Hospital, Boston, MA, USA.; Department of Genetics, Harvard Medical School, Boston, MA USA.; Tsai SQ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.; Maguire CA; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Lindsay ME; Division of Cardiology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Cardiovascular Research Center, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.; Kleinstiver BP; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.; Department of Pathology, Harvard Medical School, Boston, MA, USA.; Musolino PL; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.; Department of Neurology, Harvard Medical School, Boston, MA, USA.; Division of Neurocritical Care, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.

Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE

Vasireddy V; SwanBio Therapeutics, Inc., Philadelphia, PA, USA.; Maguire CA; Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.; Harvard Medical School, Boston, MA, USA.; Anderson DW; SwanBio Therapeutics, Inc., Philadelphia, PA, USA.; Ng C; Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.; Harvard Medical School, Boston, MA, USA.; Gong Y; Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.; Harvard Medical School, Boston, MA, USA.; Eichler F; Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.; Harvard Medical School, Boston, MA, USA.; Fourcade S; Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain.; Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain.; Guilera C; Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain.; Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain.; Onieva A; Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.; Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain.; Sanchez A; Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.; Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain.; Leal-Julià M; Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.; Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain.; Verdés S; Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.; Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain.; Dijkstra IME; Laboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, the Netherlands.; Kemp S; Laboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, the Netherlands.; Park H; SwanBio Therapeutics, Inc., Philadelphia, PA, USA.; Lutz T; SwanBio Therapeutics, Inc., Philadelphia, PA, USA.; Clark SW; SwanBio Therapeutics, Inc., Philadelphia, PA, USA.; Bosch A; Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.; Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain.; Centro de Investigación Biomédica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), Institudo de Salud Carlos III (ISCIII), Madrid, Spain.; Pujol A; Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain.; Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain.; Catalan Institution of Research and Advanced Studies (ICREA), 08010 Barcelona, Catalonia, Spain.; Kozarsky K; SwanBio Therapeutics, Inc., Philadelphia, PA, USA.

Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE