Article

Gene Targeted Therapies for Neurodegenerative Disorders: Strategies and Implications in ALS and SMA

2026-02-25

Abstract excerpt

Advances in technology have provided a better understanding of the genetic basis of neurodegenerative disorders and their underlying molecular pathophysiology. However, treating these disorders with conventional strategies is a major challenge. The approval of gene-targeted therapy for spinal muscular atrophy (SMA) has laid the foundation for developing therapies for other neurodegenerative disorders. Highly perso...

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Literature Corpus work: 35937628-5fed-5441-9e23-b9c067eebe1d
DOI: 10.20944/preprints202602.1463.v1

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