Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model Ameliorates α1-antitrypsin Deficiency Phenotype

α1-antitrypsin (AAT) is a circulating serine protease inhibitor secreted from the liver and important in preventing proteolytic neutrophil elastase associated tissue damage, primarily in lungs. In humans, AAT is encoded by the SERPINA1 (hSERPINA1) gene in which a point mutation (commonly referred to...

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Detalles Bibliográficos
Autores principales: Bjursell, Mikael, Porritt, Michelle J., Ericson, Elke, Taheri-Ghahfarokhi, Amir, Clausen, Maryam, Magnusson, Lisa, Admyre, Therese, Nitsch, Roberto, Mayr, Lorenz, Aasehaug, Leif, Seeliger, Frank, Maresca, Marcello, Bohlooly-Y, Mohammad, Wiseman, John
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2018
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5925576/
https://www.ncbi.nlm.nih.gov/pubmed/29500128
http://dx.doi.org/10.1016/j.ebiom.2018.02.015

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