Article

Efficient repair of human homozygous genetic mutation by CRISPR/Cas9 mediated interlocus gene conversion

2022-09-05

Abstract excerpt

DNA double-strand breaks (DSBs) induced by gene editing tools are primarily resolved either by non-homologous end joining (NHEJ) or homology-directed repair (HDR) using exogenous synthetic DNA templates. Repaired by error-prone NHEJ may lead to unexpected indels at the targeted site. In the case of most genetic disorders, HDR-mediated precise correction using an exogenous homologous sequence is ideal. However, the...

Identifiers and source

Literature Corpus work: 9170771a-4bdb-504e-912e-2b567baa3fc0
DOI: 10.1101/2022.09.05.506576

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Efficient repair of human homozygous genetic mutation by CRISPR/Cas9 mediated interlocus gene conversionDOI 10.1101/2022.09.05.506576Read

A Universal Approach to Target Various HBB Gene Mutations in Hematopoietic Stem/Progenitor Cells for Beta-Thalassemia Gene Therapy by CRISPR/Cas9 and the rAAV6 VectorSemantic proximity Read

A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease.Semantic proximity Read

Gene correction of HBB mutations in CD34+ hematopoietic stem cells using Cas9 mRNA and ssODN donorsSemantic proximity Read

Development of Gene Editing Strategies for Human <b>β</b> -Globin (HBB) Gene MutationsSemantic proximity Read

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.Semantic proximity Read

Genetic correction of concurrent α- and β-thalassemia patient-derived pluripotent stem cells by the CRISPR-Cas9 technology.Semantic proximity Read

Genome editing strategies for targeted correction of β-globin mutation in sickle cell disease: From bench to bedside.Semantic proximity Read

Development of gene editing strategies for human β-globin (HBB) gene mutations.Semantic proximity Read

Recent advancements in gene therapy for sickle cell disease and β-thalassemiaSemantic proximity Read

Clinical genome editing to treat sickle cell disease—A brief updateSemantic proximity Read

Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies.Semantic proximity Read

Homology-Directed-Repair-Based Genome Editing in HSPCs for the Treatment of Inborn Errors of Immunity and Blood DisordersSemantic proximity Read