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A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune

Matthew H Porteus1, Mara Pavel-Dinu2, Sung-Yun Pai3

  • 1Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford Medical School, Lokey Stem Cell Research Building, G3040B, MC 5462, 265 Campus Drive, Stanford, CA 94305, USA.

Hematology/Oncology Clinics of North America
|June 30, 2022
PubMed

Keywords:
Base editingCRISPR/Cas9DNA repairEngineered virusesGene correctionHematological diseasesHematopoietic stem cellsPrime editing

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