Article

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 Vector

2020-09-09

Abstract excerpt

<h4>Background: </h4> Engineered nuclease-mediated gene targeting through homology-directed repair (HDR) in autologous hematopoietic stem and progenitor cells (HSPCs) has the potential to cure β-thalassemia (β-thal). Although previous studies have precisely corrected site-specific HBB mutations by HDR in vitro and in vivo, targeting the various HBB mutations in β-thal is still challenging. Here, we devised a unive...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: c321e46f-5094-5327-83b5-3c996442b57a
DOI: 10.21203/rs.3.rs-68414/v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

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 VectorDOI 10.21203/rs.3.rs-68414/v1Read

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

Efficient repair of human homozygous genetic mutation by CRISPR/Cas9 mediated interlocus gene conversionSemantic proximity Read

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

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cellsSemantic proximity Read

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

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

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cellsSemantic proximity Read

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

Correction of Hemoglobin E/Beta-Thalassemia Patient-Derived iPSCs Using CRISPR/Cas9.Semantic proximity Read

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