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

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

17 Apr 2020

Abstract excerpt

Lentiviral addition of βT87Q-globin, a modified β-globin with an anti-sickling mutation, is currently being used in gene therapy trials for sickle cell disease (SCD) and β-thalassemia patients. βT87Q-globin interferes with sickle hemoglobin (HbS) polymerization. Here, we generated the SCD mutation in an immortalized human erythroid cell line (HUDEP-2) to investigate the anti-sickling activity of βT87Q-globin....

Read the complete abstract on PubMed

Topics

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

Share to Erythrocyte Function and Pathophysiology
Share to Hemoglobinopathies and Related Disorders
Share to Iron Metabolism and Disorders

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

Source

Identifiers

PMID: 32405513
DOI: 10.1016/j.omtm.2020.04.013

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 23 Oct 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

βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid CellsPMID 32405513Read ↗

Gene Therapy in a Patient with Sickle Cell DiseaseSemantic 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 ↗

Modeling Genetic Diversity in Sickle Cell Disease Reveals Heterogeneous Responses to HbF-Inducing TherapiesSemantic proximity Read ↗

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

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β A(T87Q) - Globin GeneSemantic proximity Read ↗

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

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

Sickle cell disease: understanding pathophysiology, clinical features and advances in gene therapy approachesSemantic proximity Read ↗

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

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

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

β-globin gene transfer to human bone marrow for sickle cell diseaseSemantic proximity Read ↗

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