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Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

The robustness and safety of liver-directed gene therapy can be substantially improved by enhancing expression of the therapeutic transgene in the liver. To achieve this, we developed a new approach of rational in silico vector design. This approach relies on a genome-wide bio-informatics strategy t...

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Detalles Bibliográficos
Autores principales: Chuah, Marinee K, Petrus, Inge, De Bleser, Pieter, Le Guiner, Caroline, Gernoux, Gwladys, Adjali, Oumeya, Nair, Nisha, Willems, Jessica, Evens, Hanneke, Rincon, Melvin Y, Matrai, Janka, Di Matteo, Mario, Samara-Kuko, Ermira, Yan, Bing, Acosta-Sanchez, Abel, Meliani, Amine, Cherel, Ghislaine, Blouin, Véronique, Christophe, Olivier, Moullier, Philippe, Mingozzi, Federico, VandenDriessche, Thierry
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4435486/
https://www.ncbi.nlm.nih.gov/pubmed/24954473
http://dx.doi.org/10.1038/mt.2014.114