Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells

Monogenic disorders of the blood system have the potential to be treated by autologous stem cell transplantation of ex vivo genetically modified hematopoietic stem and progenitor cells (HSPCs). The sgRNA/Cas9 system allows for precise modification of the genome at single nucleotide resolution. Howev...

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Detalles Bibliográficos
Autores principales: Benitez, Elizabeth K., Lomova Kaufman, Anastasia, Cervantes, Lilibeth, Clark, Danielle N., Ayoub, Paul G., Senadheera, Shantha, Osborne, Kyle, Sanchez, Julie M., Crisostomo, Ralph Valentine, Wang, Xiaoyan, Reuven, Nina, Shaul, Yosef, Hollis, Roger P., Romero, Zulema, Kohn, Donald B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Genome Editing
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8525354/
https://www.ncbi.nlm.nih.gov/pubmed/34713224
http://dx.doi.org/10.3389/fgeed.2020.601541

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