CRISPR correction of a homozygous low‐density lipoprotein receptor mutation in familial hypercholesterolemia induced pluripotent stem cells

Familial hypercholesterolemia (FH) is a hereditary disease primarily due to mutations in the low‐density lipoprotein receptor (LDLR) that lead to elevated cholesterol and premature development of cardiovascular disease. Homozygous FH patients (HoFH) with two dysfunctional LDLR alleles are not as suc...

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Detalles Bibliográficos
Autores principales: Omer, Linda, Hudson, Elizabeth A., Zheng, Shirong, Hoying, James B., Shan, Yuan, Boyd, Nolan L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2017
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5677509/
https://www.ncbi.nlm.nih.gov/pubmed/29130076
http://dx.doi.org/10.1002/hep4.1110

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5677509/
https://www.ncbi.nlm.nih.gov/pubmed/29130076
http://dx.doi.org/10.1002/hep4.1110

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