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Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia

Hereditary persistence of fetal hemoglobin (HPFH) is a condition in some individuals who have a high level of fetal hemoglobin throughout life. Individuals with compound heterozygous β-thalassemia or sickle cell disease (SCD) and HPFH have milder clinical manifestations. Using RNA-guided clustered r...

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Publicat a:Proc Natl Acad Sci U S A
Autors principals: Ye, Lin, Wang, Jiaming, Tan, Yuting, Beyer, Ashley I., Xie, Fei, Muench, Marcus O., Kan, Yuet Wai
Format: Artigo
Idioma:Inglês
Publicat: National Academy of Sciences 2016
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5035856/
https://ncbi.nlm.nih.gov/pubmed/27601644
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1612075113
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