Article

High-level correction of the sickle mutation amplified <i>in vivo</i> during erythroid differentiation

2018-10-03

Abstract excerpt

<h4>ABSTRACT</h4> Sickle Cell Disease (SCD), one of the world’s most common genetic disorders, causes anemia and progressive multiorgan damage that typically shortens lifespan by decades; currently there is no broadly applicable curative therapy. Here we show that Cas9 RNP-mediated gene editing with an ssDNA oligonucleotide donor yields markerless correction of the sickle mutation in more than 30% of long-term en...

Topics

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

Identifiers and source

Literature Corpus work: 38773641-dc7f-5939-9665-a75dce9116dc
DOI: 10.1101/432716

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

High-level correction of the sickle mutation amplified <i>in vivo</i> during erythroid differentiationDOI 10.1101/432716Read

High-level correction of the sickle mutation is amplified in vivo during erythroid differentiationSemantic proximity Read

Efficient Correction of the Sickle Mutation in Human Hematopoietic Stem Cells Using a Cas9 Ribonucleoprotein ComplexSemantic proximity Read

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells.Semantic proximity Read

Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell diseaseSemantic proximity Read

Gene editing without <i>ex vivo</i> culture evades genotoxicity in human hematopoietic stem cellsSemantic proximity Read

Evaluation of persistence and fate of <i>ex vivo</i> edited-HSC modified with donor template and Its role in correcting Sickle Cell DiseaseSemantic proximity Read

Enhanced γ-globin reactivation and sickle cell correction through a repressor-to-activator motif switch in the <i>HBG1/2</i> promotersSemantic proximity Read

Revolutionising healing: Gene Editing's breakthrough against sickle cell diseaseSemantic proximity Read

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

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells.Semantic proximity Read

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

Therapeutic adenine base editing of human hematopoietic stem cellsSemantic proximity Read