Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care

Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care

Gene therapy holds promise as a life-changing option for individuals with genetic variants that give rise to disease. FDA-approved gene therapies for Spinal Muscular Atrophy (SMA), cerebral adrenoleukodystrophy, β-Thalassemia, hemophilia A/B, retinal dystrophy, and Duchenne Muscular Dystrophy have g...

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Bibliographic Details
Main Authors: Marian L. Henderson, Jacob K. Zieba, Xiaopeng Li, Daniel B. Campbell, Michael R. Williams, Daniel L. Vogt, Caleb P. Bupp, Yvonne M. Edgerly, Surender Rajasekaran, Nicholas L. Hartog, Jeremy W. Prokop, Jena M. Krueger
Format: Article
Language:English
Published: MDPI AG 2024-01-01
Series:BioTech
Subjects:
gene therapy
genetic syndromes
clinical trials
Online Access:https://www.mdpi.com/2673-6284/13/1/1

Internet

https://www.mdpi.com/2673-6284/13/1/1

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