Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis

Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis

Spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS) are severe nervous system diseases characterized by the degeneration of lower motor neurons. They share a number of additional pathological, cellular, and genetic parallels suggesting that mechanistic and clinical insights into on...

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Manylion Llyfryddiaeth
Prif Awduron: Andrew P. Tosolini, James N. Sleigh
Fformat: Erthygl
Iaith:English
Cyhoeddwyd: Frontiers Media S.A. 2017-12-01
Cyfres:Frontiers in Molecular Neuroscience
Pynciau:
adeno-associated virus (AAV)
ALS
antisense oligonucleotide (ASO)
motor neuron disease (MND)
neurodegeneration
neurotrophic factor
Mynediad Ar-lein:http://journal.frontiersin.org/article/10.3389/fnmol.2017.00405/full

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http://journal.frontiersin.org/article/10.3389/fnmol.2017.00405/full

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