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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Библиографические подробности
Опубликовано в: :EBioMedicine
Главные авторы: 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
Формат: Artigo
Язык:Inglês
Опубликовано: Elsevier 2018
Предметы:
Research Paper
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC5925576/
https://ncbi.nlm.nih.gov/pubmed/29500128
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ebiom.2018.02.015
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