AAV9-DOK7 gene therapy reduces disease severity in Smn SMA model mice-Reference-Cited by-同舟云学术

AAV9-DOK7 gene therapy reduces disease severity in Smn SMA model mice

Published:2020-09 Issue:1 Volume:530 Page:107-114
ISSN:0006-291X
Container-title:Biochemical and Biophysical Research Communications
language:en
Short-container-title:Biochemical and Biophysical Research Communications

Author:

Kaifer Kevin A.,Villalón Eric^ORCID,Smith Caley E.^ORCID,Simon Madeline E.,Marquez Jose,Hopkins Abigail E.,Morcos Toni I.^ORCID,Lorson Christian L.^ORCID

Funder

NIH

NIH Training

NIH PREP

Publisher

Elsevier BV

Subject

Cell Biology,Molecular Biology,Biochemistry,Biophysics

Reference51 articles.

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3. SMN in spinal muscular atrophy and snRNP biogenesis;Coady;Adv. Rev.,2011

4. Spinal muscular atrophy: the role of SMN in axonal mRNA regulation;Fallini;Brain Res.,2012

5. Nusinersen: a review in 5q spinal muscular atrophy;Hoy;CNS Drugs,2018

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