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Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges

Version 1 : Received: 28 June 2024 / Approved: 28 June 2024 / Online: 29 June 2024 (06:33:01 CEST)

A peer-reviewed article of this Preprint also exists.

Haque, U.S.; Yokota, T. Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges. Genes 2024, 15, 999. Haque, U.S.; Yokota, T. Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges. Genes 2024, 15, 999.

Abstract

Keywords: spinal muscular atrophy (SMA); survival of motor neuron 1 (SMN1); SMN2; SMN protein; antisense oligonucleotide (ASO); nusinersen; gene therapy; onasemnogene; risdiplam; small molecule, combination therapy

Keywords

spinal muscular atrophy (SMA); survival of motor neuron 1 (SMN1); SMN2; SMN protein; antisense oligonucleotide (ASO); nusinersen; gene therapy; onasemnogene; risdiplam; small molecule; combination therapy

Subject

Biology and Life Sciences, Neuroscience and Neurology

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