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

Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Sept 2021

Nualkaew Tiwaporn, Sii-Felice Karine, Giorgi Marie, McColl Bradley, Gouzil Julie, Glaser Astrid, Voon Hsiao P J, Tee Hsin Y, Grigoriadis George, Svasti Saovaros, Fucharoen Suthat, Hongeng Suradej, Leboulch Philippe, Payen Emmanuel, Vadolas Jim

Abstract excerpt

A primary challenge in lentiviral gene therapy of β-hemoglobinopathies is to maintain low vector copy numbers to avoid genotoxicity while being reliably therapeutic for all genotypes. We designed a high-titer lentiviral vector, LVβ-shα2, that allows coordinated expression of the therapeutic βA-T87Q-globin gene and of an intron-embedded miR-30-based short hairpin RNA (shRNA) selectively targeting the α2-globin...

Read the complete abstract on PubMed

Topics

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

Share to alpha-Globins
Share to beta-Globins

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to beta-Thalassemia

Share to Erythroid Cells

Share to K562 Cells

Share to Lentivirus

Share to Primary Cell Culture

Share to RNA, Small Interfering

Source

Open in PubMed Open full text

Identifiers

PMID: 33940155
DOI: 10.1016/j.ymthe.2021.04.037
PMCID: PMC8417505

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia.PMID 33940155Read ↗

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

Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by HBBAS3 Gene Addition and Mutation-Specific RNA Interference.Semantic proximity Read ↗

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

Manu on Lenti G2B Gene Therapy for β-ThalassemiaSemantic proximity Read ↗

Gene Therapy for Hemoglobinopathies: Tremendous Successes and Remaining CaveatsSemantic proximity Read ↗

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

Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia.Semantic proximity Read ↗

The Future of Gene Therapy for Transfusion-Dependent Beta-Thalassemia: The Power of the Lentiviral Vector for Genetically Modified Hematopoietic Stem CellsSemantic proximity Read ↗

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

Gene therapy of hemoglobinopathies: progress and future challengesSemantic proximity Read ↗

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.Semantic proximity Read ↗

Restoration of the balanced alpha/beta-globin gene expression in beta654-thalassemia mice using combined RNAi and antisense RNA approach.Semantic proximity Read ↗

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