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Preserved in Portico This version is not peer-reviewed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
Copyright: This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
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