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

Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.

Human gene therapy - 1 Oct 2018

Urbinati Fabrizia, Campo Fernandez Beatriz, Masiuk Katelyn E, Poletti Valentina, Hollis Roger P, Koziol Colin, Kaufman Michael L, Brown Devin, Mavilio Fulvio, Kohn Donald B

Abstract excerpt

Sickle cell disease (SCD) is an inherited blood disorder caused by a single amino acid substitution in the β-globin chain of hemoglobin. Gene therapy is a promising therapeutic alternative, particularly in patients lacking an allogeneic bone marrow (BM) donor. One of the major challenges for an effective gene therapy approach is the design of an efficient vector that combines high-level and long-term β-globin...

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 beta-Globins

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Colony-Forming Units Assay

Share to Gene Order

Share to Genetic Therapy

Share to Hematopoietic Stem Cells

Share to Lentivirus

Share to Transduction, Genetic

Source

Identifiers

PMID: 30198339
DOI: 10.1089/hum.2018.061

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.PMID 30198339Read ↗

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

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

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

Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region ElementsSemantic proximity Read ↗

Gene therapy for hemoglobinopathies: progress and challenges.Semantic proximity Read ↗

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

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

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

Diverse Approaches to Gene Therapy of Sickle Cell DiseaseSemantic proximity Read ↗

Investigational curative gene therapy approaches to sickle cell diseaseSemantic proximity Read ↗

Gene Therapy for β-Hemoglobinopathies.Semantic proximity Read ↗

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

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