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

3 Jan 2024

Abstract excerpt

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 generated buzz around the ability to change the course of genetic syndromes. However, this excitement risks...

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PMID: 38247731
DOI: 10.3390/biotech13010001

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