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Adeno-Associated Virus Gene Repair Corrects a Mouse Model of Hereditary Tyrosinemia In Vivo

Adeno-associated virus (AAV) vectors are ideal for performing gene repair due to their ability to target multiple different genomic loci, low immunogenicity, capability to achieve targeted and stable expression through integration, and low mutagenic and oncogenic potential. However, many handicaps t...

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Autors principals: Paulk, Nicole K., Wursthorn, Karsten, Wang, Zhongya, Finegold, Milton J., Kay, Mark A., Grompe, Markus
Format: Artigo
Idioma:Inglês
Publicat: 2010
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3136243/
https://ncbi.nlm.nih.gov/pubmed/20162619
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/hep.23481
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