Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy-Reference-Cited by-同舟云学术

Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy

Published:2021-06-01 Issue:11-12 Volume:32 Page:563-580
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Rosenberg Jonathan B.¹,Chen Alvin¹,De Bishnu P.¹,Dyke Jonathan P.²,Ballon Douglas J.²,Monette Sebastien³,Ricart Arbona Rodolfo J.³,Kaminsky Stephen M.¹,Crystal Ronald G.¹,Sondhi Dolan¹

Affiliation:

1. Department of Genetic Medicine, Weill Cornell Medical College, New York, New York, USA

2. Department of Radiology, Citigroup Biomedical Imaging Center, Weill Cornell Medical College, New York, New York, USA

3. Center for Comparative Medicine and Pathology, Memorial Sloan Kettering Cancer Center and Weill Cornell Medicine, New York, New York, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2020.269

Reference62 articles.

1. Developing treatment options for metachromatic leukodystrophy

2. Gene therapy for metachromatic leukodystrophy

3. Peripheral neuropathy in metachromatic leukodystrophy: current status and future perspective

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