Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease-Reference-Cited by-同舟云学术

Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease

Published:2021-02 Issue:2 Volume:29 Page:691-701
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Li Yedda,Miller Christopher A.,Shea Lauren K.,Jiang Xuntian,Guzman Miguel A.,Chandler Randy J.,Ramakrishnan Sai M.,Smith Stephanie N.,Venditti Charles P.,Vogler Carole A.,Ory Daniel S.,Ley Timothy J.,Sands Mark S.

Funder

International Foundation for Research in Paraplegia

National Institutes of Health

National Human Genome Research Institute

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

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5. The Twitcher mouse: an enzymatically authentic model of human globoid cell leukodystrophy (Krabbe disease);Kobayashi;Brain Res.,1980

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