Article
The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells.
Nature communications - 20 Jan 2021
Sharma Rajiv, Dever Daniel P, Lee Ciaran M, Azizi Armon, Pan Yidan, Camarena Joab, Köhnke Thomas, Bao Gang, Porteus Matthew H, Majeti Ravindra
Abstract excerpt
Targeted DNA correction of disease-causing mutations in hematopoietic stem and progenitor cells (HSPCs) may enable the treatment of genetic diseases of the blood and immune system. It is now possible to correct mutations at high frequencies in HSPCs by combining CRISPR/Cas9 with homologous DNA donors. Because of the precision of gene correction, these approaches preclude clonal tracking of gene-targeted HSPCs....
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