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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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Detalles Bibliográficos
Autores principales:	Stephens, Calvin J., Kashentseva, Elena, Everett, William, Kaliberova, Lyudmila, Curiel, David T.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2018
Materias:	Brief Communication
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5919923/ https://www.ncbi.nlm.nih.gov/pubmed/29588497 http://dx.doi.org/10.1038/s41434-018-0003-1

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