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...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
Assuntos:
Original Article
Acesso em linha: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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