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Gene Therapy for Rare Neurological Disorders.
Flotte TR, Gessler DJ. Gene Therapy for Rare Neurological Disorders. Clin Pharmacol Ther. 2022 04; 111(4):743-757.
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PubMed
subject areas
Gene Editing
Genetic Therapy
Genetic Vectors
Humans
Muscular Atrophy, Spinal
Mutation
authors with profiles
Terence R Flotte MD
Dominic J Gessler MD, PhD