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Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9

Serum deficiency diseases such as alpha-1-antitrypsin deficiency are characterized by reduced function of serum proteins, caused by deleterious genetic mutations. These diseases are promising targets for genetic interventions. Gene therapies using viral vectors have been used to introduce correct co...

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Bibliografiske detaljer
Udgivet i:Gene Ther
Main Authors: Stephens, Calvin J., Kashentseva, Elena, Everett, William, Kaliberova, Lyudmila, Curiel, David T.
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2018
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5919923/
https://ncbi.nlm.nih.gov/pubmed/29588497
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41434-018-0003-1
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