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Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

Sickle cell disease (SCD) is characterized by a single point mutation in the seventh codon of the β-globin gene. Site-specific correction of the sickle mutation in hematopoietic stem cells would allow for permanent production of normal red blood cells. Using zinc-finger nucleases (ZFNs) designed to...

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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Blood
Egile Nagusiak: Hoban, Megan D., Cost, Gregory J., Mendel, Matthew C., Romero, Zulema, Kaufman, Michael L., Joglekar, Alok V., Ho, Michelle, Lumaquin, Dianne, Gray, David, Lill, Georgia R., Cooper, Aaron R., Urbinati, Fabrizia, Senadheera, Shantha, Zhu, Allen, Liu, Pei-Qi, Paschon, David E., Zhang, Lei, Rebar, Edward J., Wilber, Andrew, Wang, Xiaoyan, Gregory, Philip D., Holmes, Michael C., Reik, Andreas, Hollis, Roger P., Kohn, Donald B.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society of Hematology 2015
Gaiak:
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC4408287/
https://ncbi.nlm.nih.gov/pubmed/25733580
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2014-12-615948
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