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

Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease

1 Apr 2025

Abstract excerpt

substitution in the β-globin leads to red blood cell sickling. The transplantation of autologous, genetically modified hematopoietic stem and progenitor cells (HSPCs) is a promising treatment option for patients with SCD. We completed a Phase I/II open-label clinical trial (NCT03964792) for patients with SCD using a lentiviral vector (DREPAGLOBE) expressing a potent anti-sickling β-globin. The primary endpoint...

Read the complete abstract on PubMed

Topics

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

Share to CRISPR and Genetic Engineering
Share to Hemoglobinopathies and Related Disorders
Share to Prenatal Screening and Diagnostics

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 40169559
DOI: 10.1038/s41467-025-58321-4

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell diseasePMID 40169559Read ↗

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

Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neitherSemantic proximity Read ↗

Stem cell transplantation in sickle cell disease: therapeutic potential and challenges facedSemantic proximity Read ↗

Gene therapy for sickle cell disease: where we are now?Semantic proximity Read ↗

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

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

Advances in Sickle Cell Disease Treatment: A Comparative Review of Hematopoietic Stem Cell Transplantation and Gene Therapy (Casgevy and Lyfgenia)Semantic proximity Read ↗

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

Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB ‐206 studySemantic proximity Read ↗

Current state of gene therapy in sickle cell diseaseSemantic proximity Read ↗

Sickle Cell Disease: From Genetics to Curative ApproachesSemantic proximity Read ↗

Sickle Cell DiseaseSemantic proximity Read ↗

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