Adeno-Associated Virus-Based Gene Therapy for Lifelong Correction of Genetic Disease
Author:
Affiliation:
1. Program in Molecular Medicine, University of Iowa, Iowa City, Iowa, USA.
2. Department of Pediatrics, University of Iowa, Iowa City, Iowa, USA.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.liebertpub.com/doi/pdf/10.1089/hum.2020.138
Reference112 articles.
1. AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
2. Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
3. Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
4. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
5. Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
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