AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity

The gene therapy field has been galvanized by two technologies that have revolutionized treating genetic diseases: vectors based on adeno-associated viruses (AAVs), and clustered regularly interspaced short palindromic repeats (CRISPR)-Cas gene-editing tools. When combined into one platform, these s...

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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Tran, Ngoc Tam, Heiner, Cheryl, Weber, Kristina, Weiand, Michael, Wilmot, Daniella, Xie, Jun, Wang, Dan, Brown, Alexander, Manokaran, Sangeetha, Su, Qin, Zapp, Maria L., Gao, Guangping, Tai, Phillip W.L.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2020
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7397707/
https://ncbi.nlm.nih.gov/pubmed/32775498
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.07.007
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