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

A simplified G-CSF–free procedure allows for in vivo HSC gene therapy of sickle cell disease in a mouse model

6 Jun 2024

Abstract excerpt

ABSTRACT: We have reported the direct repair of the sickle cell mutation in vivo in a disease model using vectorized prime editors after hematopoietic stem cell (HSC) mobilization with granulocyte colony-stimulating factor (G-CSF)/AMD3100. The use of G-CSF for HSC mobilization is a hurdle for the clinical translation of this approach. Here, we tested a G-CSF-free mobilization regimen using WU-106, an inhibitor of...

Read the complete abstract on PubMed

Topics

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

Share to Parvovirus B19 Infection Studies
Share to RNA Interference and Gene Delivery
Share to Virus-based gene therapy research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 38843380
DOI: 10.1182/bloodadvances.2024012757

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

A simplified G-CSF–free procedure allows for in vivo HSC gene therapy of sickle cell disease in a mouse modelPMID 38843380Read ↗

In vivo HSC prime editing rescues Sickle Cell Disease in a mouse modelSemantic proximity Read ↗

Introducing a hemoglobin G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in mice.Semantic proximity Read ↗

Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.Semantic proximity Read ↗

Advances in Gene Therapy for Sickle Cell Disease: From Preclinical Innovations to Clinical Implementation and Access ChallengesSemantic proximity Read ↗

Genetic therapies for the first molecular diseaseSemantic proximity Read ↗

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

Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in miceSemantic proximity Read ↗

Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell DiseaseSemantic proximity Read ↗

Treating Sickle Cell Disease: Gene Therapy ApproachesSemantic proximity Read ↗

Cas9-AAV6 Gene Correction of Beta-Globin in Autologous HSCs Improves Sickle Cell Disease Erythropoiesis in MiceSemantic proximity Read ↗

Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in miceSemantic proximity Read ↗

In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathiesSemantic proximity Read ↗

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