Sustained Knockdown of a Disease-Causing Gene in Patient-Specific Induced Pluripotent Stem Cells Using Lentiviral Vector-Based Gene Therapy

Patient-specific induced pluripotent stem cells (iPSCs) hold great promise for studies on disease-related developmental processes and may serve as an autologous cell source for future treatment of many hereditary diseases. New genetic engineering tools such as zinc finger nucleases and transcription...

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Autors principals: Eggenschwiler, Reto, Loya, Komal, Wu, Guangming, Sharma, Amar Deep, Sgodda, Malte, Zychlinski, Daniela, Herr, Christian, Steinemann, Doris, Teckman, Jeffrey, Bals, Robert, Ott, Michael, Schambach, Axel, Schöler, Hans Robert, Cantz, Tobias
Format: Artigo
Idioma:Inglês
Publicat: AlphaMed Press 2013
Matèries:
Embryonic Stem Cells/Induced Pluripotent Stem (iPS) Cells
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3754465/
https://ncbi.nlm.nih.gov/pubmed/23926210
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.5966/sctm.2013-0017
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