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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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Bibliographische Detailangaben
Hauptverfasser: Paulk, Nicole K., Wursthorn, Karsten, Wang, Zhongya, Finegold, Milton J., Kay, Mark A., Grompe, Markus
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2010
Schlagworte:
Online Zugang: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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