Translational Medicine: Evolution, Fulfillment, and Belief in Gene Therapy
Author:
Affiliation:
1. Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio
2. Department of Pediatrics and Neurology, The Ohio State University, Columbus, Ohio.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.29103.jrm
Reference30 articles.
1. T Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years
2. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial
3. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
4. Duchenne Muscular Dystrophy: Functional Ischemia Reproduces Its Characteristic Lesions
5. Proximal Myopathy induced by 5-HT-Imipramine simulates Duchenne Dystrophy
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1. The Hidden Disease: Delayed Diagnosis in Duchenne Muscular Dystrophy and Co-Occurring Conditions;Journal of Developmental & Behavioral Pediatrics;2022-07-06
2. Translating molecular therapies for spinal muscular atrophy;Neurotherapeutics in the Era of Translational Medicine;2021
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