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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Príomhchruthaitheoirí: Andrew P. Tosolini, James N. Sleigh
Formáid: Alt
Teanga:English
Foilsithe / Cruthaithe: Frontiers Media S.A. 2017-12-01
Sraith:Frontiers in Molecular Neuroscience
Ábhair:
adeno-associated virus (AAV)
ALS
antisense oligonucleotide (ASO)
motor neuron disease (MND)
neurodegeneration
neurotrophic factor
Rochtain ar líne:http://journal.frontiersin.org/article/10.3389/fnmol.2017.00405/full

Ar líne

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

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