ArticleMolecular therapy. Methods & clinical development2025
Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2.
Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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Who cites it
4 citing papers in PubMed, 1 synthesis or guideline pooled it.
- A therapeutic atlas of monogenic inflammatory bowel disease.Journal of Crohn's & colitis · 2026Pooled it
- Adenosine deaminase type 2 deficiency: From rare to common.Journal of human immunity · 2026Review
- DADA2 as a Model of Monogenic Immune Vasculopathy: From Immunopathogenesis to Precision Therapeutics.Biomolecules · 2026Review
- Opinion: Why paediatric rheumatologists need to understand inborn errors of immunity.Frontiers in pediatrics · 2025Article
Corrections and comments
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Authors and funding
28 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Deficiency of adenosine deaminase 2 (DADA2) is a monogenic autoinflammatory disorder characterized by systemic inflammation, vasculopathy, immunodeficiency, and bone marrow failure. Current therapies-including anti-TNF agents and allogeneic hematopoietic stem cell transplantation (HSCT)-have limitations, especially for patients with hematologic involvement or no matched donor. We developed a lentiviral vector (LV.ADA2) to restore ADA2 expression in patient hematopoietic stem and progenitor cells (HSPCs) and evaluated its safety and efficacy in preclinical models. LV.ADA2 transduction of mobilized peripheral blood HSPCs from healthy donors resulted in stable ADA2 expression and secretion without impairing clonogenicity, multilineage differentiation, or long-term engraftment in immunodeficient mice. In HSPCs from 12 DADA2 patients, LV.ADA2 restored ADA2 protein and enzymatic activity preserving colony-forming ability and multilineage differentiation
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.