RNAi prevents and reverses phenotypes induced by mutant human ataxin‐1-Reference-Cited by-同舟云学术

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RNAi prevents and reverses phenotypes induced by mutant human ataxin‐1

Published:2016-11 Issue:5 Volume:80 Page:754-765
ISSN:0364-5134
Container-title:Annals of Neurology
language:en
Short-container-title:Ann Neurol.

Author:

Keiser Megan S.¹,Monteys Alejandro Mas¹,Corbau Romuald¹²,Gonzalez‐Alegre Pedro¹³,Davidson Beverly L.¹⁴

Affiliation:

1. Raymond G. Perelman Center for Cellular and Molecular TherapeuticsChildren's Hospital of PhiladelphiaPhiladelphia PA

2. Spark TherapeuticsPhiladelphia PA

3. Department of NeurologyUniversity of PennsylvaniaPhiladelphia PA

4. Department of Pathology and Laboratory MedicineUniversity of PennsylvaniaPhiladelphia PA

Funder

NIH National Institute of Neurological Disorders and Stroke

National Ataxia Foundation Pioneer Award

Children's Hospital of Philadelphia Research Institute

Publisher

Wiley

Subject

Neurology (clinical),Neurology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/ana.24789

Reference29 articles.

1. Trinucleotide Repeat Disorders

2. SCA1 transgenic mice: A model for neurodegeneration caused by an expanded CAG trinucleotide repeat

3. Purkinje Cell Expression of a Mutant Allele ofSCA1in Transgenic Mice Leads to Disparate Effects on Motor Behaviors, Followed by a Progressive Cerebellar Dysfunction and Histological Alterations

4. Recovery from Polyglutamine-Induced Neurodegeneration in Conditional SCA1 Transgenic Mice

5. RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia

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