MECP2 Isoform-Specific Vectors with Regulated Expression for Rett Syndrome Gene Therapy

BACKGROUND: Rett Syndrome (RTT) is an Autism Spectrum Disorder and the leading cause of mental retardation in females. RTT is caused by mutations in the Methyl CpG-Binding Protein-2 (MECP2) gene and has no treatment. Our objective is to develop viral vectors for MECP2 gene transfer into Neural Stem...

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Main Authors: Rastegar, Mojgan, Hotta, Akitsu, Pasceri, Peter, Makarem, Maisam, Cheung, Aaron Y. L., Elliott, Shauna, Park, Katya J., Adachi, Megumi, Jones, Frederick S., Clarke, Ian D., Dirks, Peter, Ellis, James
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2009
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2728539/
https://ncbi.nlm.nih.gov/pubmed/19710912
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0006810
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