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

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

Nature - 17 Nov 2016

Dever Daniel P, Bak Rasmus O, Reinisch Andreas, Camarena Joab, Washington Gabriel, Nicolas Carmencita E, Pavel-Dinu Mara, Saxena Nivi, Wilkens Alec B, Mantri Sruthi, Uchida Nobuko, Hendel Ayal, Narla Anupama, Majeti Ravindra, Weinberg Kenneth I, Porteus Matthew H

Abstract excerpt

The β-haemoglobinopathies, such as sickle cell disease and β-thalassaemia, are caused by mutations in the β-globin (HBB) gene and affect millions of people worldwide. Ex vivo gene correction in patient-derived haematopoietic stem cells followed by autologous transplantation could be used to cure β-haemoglobinopathies. Here we present a CRISPR/Cas9 gene-editing system that combines Cas9 ribonucleoproteins and...

Read the complete abstract on PubMed

Topics

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

Share to Anemia, Sickle Cell
Share to Antigens, CD34

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Share to beta-Globins

Share to CRISPR-Associated Proteins

Share to Dependovirus

Share to Homologous Recombination

Share to Mice, Inbred NOD

Share to Microspheres

Source

Open in PubMed Open full text

Identifiers

PMID: 27820943
DOI: 10.1038/nature20134
PMCID: PMC5898607

Genes

HBB

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence. Graph snapshot: 2026-09-29.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.PMID 27820943Read ↗

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

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

A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.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 correction of HBB mutations in CD34+ hematopoietic stem cells using Cas9 mRNA and ssODN donorsSemantic proximity Read ↗

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

Advances in Gene Therapy for Inherited HaemoglobinopathiesSemantic proximity Read ↗

Gene Therapy for β-Haemoglobinopathies: From Molecular Correction to Curative MedicineSemantic proximity Read ↗

Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemiaSemantic proximity Read ↗

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

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