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

β-globin gene transfer to human bone marrow for sickle cell disease

30 Jun 2013

Abstract excerpt

Autologous hematopoietic stem cell gene therapy is an approach to treating sickle cell disease (SCD) patients that may result in lower morbidity than allogeneic transplantation. We examined the potential of a lentiviral vector (LV) (CCL-βAS3-FB) encoding a human hemoglobin (HBB) gene engineered to impede sickle hemoglobin polymerization (HBBAS3) to transduce human BM CD34+ cells from SCD donors and prevent...

Read the complete abstract on PubMed

Topics

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

Share to Hemoglobinopathies and Related Disorders
Share to Parvovirus B19 Infection Studies
Share to Prenatal Screening and Diagnostics

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 23863630
DOI: 10.1172/jci67930

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

β-globin gene transfer to human bone marrow for sickle cell diseasePMID 23863630Read ↗

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease PhenotypeSemantic proximity Read ↗

βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid CellsSemantic proximity Read ↗

Combination of lentiviral and genome editing technologies for the treatment of sickle cell diseaseSemantic proximity Read ↗

Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.Semantic proximity Read ↗

Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical TrialsSemantic proximity Read ↗

Gene Therapy in a Patient with Sickle Cell DiseaseSemantic proximity Read ↗

Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategiesSemantic proximity Read ↗

Gene therapy for sickle cell disease: moving from the bench to the bedsideSemantic proximity Read ↗

Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategiesSemantic proximity Read ↗

Recent advances in globin gene transfer for the treatment of beta-thalassemia and sickle cell anemiaSemantic proximity Read ↗

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene TherapySemantic proximity Read ↗

Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell diseaseSemantic proximity Read ↗

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