Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates

Site-specific correction of a point mutation causing a monogenic disease in autologous hematopoietic stem and progenitor cells (HSPCs) can be used as a treatment of inherited disorders of the blood cells. Sickle cell disease (SCD) is an ideal model to investigate the potential use of gene editing to...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Mol Ther
Egile Nagusiak: Romero, Zulema, Lomova, Anastasia, Said, Suzanne, Miggelbrink, Alexandra, Kuo, Caroline Y., Campo-Fernandez, Beatriz, Hoban, Megan D., Masiuk, Katelyn E., Clark, Danielle N., Long, Joseph, Sanchez, Julie M., Velez, Miriam, Miyahira, Eric, Zhang, Ruixue, Brown, Devin, Wang, Xiaoyan, Kurmangaliyev, Yerbol Z., Hollis, Roger P., Kohn, Donald B.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society of Gene & Cell Therapy 2019
Gaiak:
Original Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC6697408/
https://ncbi.nlm.nih.gov/pubmed/31178391
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.05.014
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