Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates

23 May 2019

Abstract excerpt

Site-specific correction of a point mutation causing a monogenic disease in autologous hematopoietic stem and progenitor cells (HSPCs) can be used as a treatment of inherited disorders of the blood cells. Sickle cell disease (SCD) is an ideal model to investigate the potential use of gene editing to transvert a single point mutation at the β-globin locus (HBB). We compared the activity of zinc-finger nucleases...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to CRISPR and Genetic Engineering
Share to Mosquito-borne diseases and control
Share to Virus-based gene therapy research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 31178391
DOI: 10.1016/j.ymthe.2019.05.014

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor TemplatesPMID 31178391Read ↗

In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery MethodsSemantic proximity Read ↗

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.Semantic proximity Read ↗

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

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

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

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

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

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

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

⌁Select a neighboring publication to make it the new centre.