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Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice.

Document Type

Article

Author

van Til, Niek P.; Dogan, Yildirim; Barese, Cecilia; Unnisa, Zeenath; Guda, Swaroopa; Schindler, Rena; Yoon, John; Jacobs, Mary; Chiyyeadu, Abhishek; Pizzurro, Daniella; Liu, Bianling; Braun, Maurine; Oborski, Christine; Schambach, Axel; Harper, Claudia; Pfeifer, Richard; Mason, Chris

Source

Molecular Genetics & Metabolism. Feb2021, Vol. 132 Issue 2, pS107-S107. 1p.

Subject

*HEMATOPOIETIC stem cells
*STEM cell treatment
*NEUROMUSCULAR transmission
*GLYCOGEN storage disease type II
*MICE
*CENTRAL nervous system

Language

ISSN

1096-7192

Online Access

Full Text (ScienceDirect 종량제) Web of Science JCR 저널정보 Scopus Find it@PNU

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