Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome-Reference-Cited by-同舟云学术

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Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome

Published:2017-06 Issue: Volume:5 Page:180-190
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Gadalla Kamal K.E.,Vudhironarit Thishnapha,Hector Ralph D.,Sinnett Sarah,Bahey Noha G.,Bailey Mark E.S.,Gray Steven J.,Cobb Stuart R.

Funder

Rett Syndrome Research Trust

Chief Scientist Office

NIH

Rett Syndrome Association Scotland

R.S. MacDonald Charitable Trust

Stoneygate Trust

Rosetrees Trust

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference45 articles.

1. Rett syndrome: revised diagnostic criteria and nomenclature;Neul;Ann. Neurol.,2010

2. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2;Amir;Nat. Genet.,1999

3. Molecular genetics of Rett syndrome: when DNA methylation goes unrecognized;Bienvenu;Nat. Rev. Genet.,2006

4. Rett syndrome: a complex disorder with simple roots;Lyst;Nat. Rev. Genet.,2015

5. Deficiency of methyl-CpG binding protein-2 in CNS neurons results in a Rett-like phenotype in mice;Chen;Nat. Genet.,2001

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