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Gene therapy in rare diseases: The benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID

Document Type

Article

Author

Stirnadel-Farrant, H.; Garman, N.; Appleby, J.; Kudari, M.; Chopra, B.; Batta, R.; Ng, P.; Imrie, J.; Giannelli, S.; Gabaldo, M.; Corti, A.; Zancan, S.; Aiuti, A.; Cicalese, M.P.; Davinelli, M.

Source

In: Orphanet Journal of Rare Diseases. (Orphanet Journal of Rare Diseases, 6 April 2018, 13(1))

Subject

Language

English

ISSN

17501172

Online Access

EBSCOHost PDF Full Text (ProQuest Central) Full Text (Gale Academic Onefile) Open Access (BioMed Central) Web of Science JCR 저널정보 Scopus Find it@PNU

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