Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice

BACKGROUND: Synthetic zinc finger (ZF) proteins can be targeted to desired DNA sequences and are useful tools for gene therapy. We recently developed a ZF transcription repressor (ZF-KOX1) able to bind to expanded DNA CAG-repeats in the huntingtin (HTT) gene, which are found in Huntington’s disease...

Descripción completa

Detalles Bibliográficos
Autores principales: Agustín-Pavón, Carmen, Mielcarek, Michal, Garriga-Canut, Mireia, Isalan, Mark
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2016
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5013590/
https://www.ncbi.nlm.nih.gov/pubmed/27600816
http://dx.doi.org/10.1186/s13024-016-0128-x

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5013590/
https://www.ncbi.nlm.nih.gov/pubmed/27600816
http://dx.doi.org/10.1186/s13024-016-0128-x

Ejemplares similares