Mutant Allele-Specific CRISPR Disruption in DYT1 Dystonia Fibroblasts Restores Cell Function-Reference-Cited by-同舟云学术

Mutant Allele-Specific CRISPR Disruption in DYT1 Dystonia Fibroblasts Restores Cell Function

Published:2020-09 Issue: Volume:21 Page:1-12
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Cruz Lilian,György Bence,Cheah Pike See,Kleinstiver Benjamin P.,Eimer William A.,Garcia Sara P.,Sharma Nutan,Ozelius Laurie J.,Bragg D. Cristopher,Joung J. Keith,Norberto de Souza Osmar,Macedo Timmers Luis Fernando Saraiva,Breakefield Xandra O.

Funder

NIH

Bachmann-Strauss Dystonia and Parkinson Foundation

Michael J. Fox Foundation

Tyler’s Hope for a Dystonia Cure

Edward R. and Anne G. Lefler Center

Dystonia Medical Research Foundation

Coordenação de Aperfeiçoamento de Pessoal de Nível Superior-Brasil

FAPERGS

CNPq

University of Taquari Valley

American Society of Gene & Cell Therapy

Margaret Q. Landenberger Research Foundation

Desmond and Ann Heathwood MGH Research Scholar Award

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

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3. Structures of TorsinA and its disease-mutant complexed with an activator reveal the molecular basis for primary dystonia;Demircioglu;eLife,2016

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