ReviewRegenerative therapy2025
Viral-based gene therapy clinical trials for immune deficiencies and blood disorders from 2013 until 2023 - an overview.
Review in Regenerative therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
5 citing papers in PubMed.
- Phytocompounds with immunomodulatory activities: mechanisms, therapeutic potential, and clinical prospects.Inflammopharmacology · 2026Review
- The burden of severe leukocyte adhesion deficiency, type I (LAD-I): A multiple-case study from the caregiver's perspective.Journal of human immunity · 2026Article
- Current capacity and gaps in viral vector manufacturing: a survey of centers associated with CTIWP of EBMT, NXTGEN-hightech, DARE-NL, T2EVOLVE, and GoCART coalition.Bone marrow transplantation · 2026Article
- Review
- Genomic medicine in hepatology: mechanisms and liver treatment strategies.Molecular medicine (Cambridge, Mass.) · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy (GT) as a groundbreaking approach holds promise for treating many diseases including immune deficiencies and blood disorders. GT can benefit patients suffering from these diseases, especially those without matched donors or who are at risk after hematopoietic stem cell transplantation (HSCT). Due to all the advances in the field of GT, its main challenge is still gene delivery. Generally, gene delivery systems are categorized into two types depending on utilized vectors: non-viral and viral. Viral vectors are commonly used in GT because of their high efficiency compared to non-viral vectors. In this article, all clinical trials on viral-based GT (with the exclusion of CRISPR and CAR-T cell Therapy) in the last decade for immune deficiencies and blood disorders including Severe combined immune deficiency (SCID), Wiskott-Aldrich syndrome (WAS), Chronic granulomatous disease (CGD), Leukocyte adhesion deficiency (LAD), Fanconi anemia (FA), Hemoglobinopathies, and Hemophilia will thoroughly be discussed. Moreover, viral vectors used in these trials including Retroviruses (RVs), Lentiviruses (LVs), and Adeno-Associated Viruses (AAVs) will be reviewed. This review provides a concise overview of traditional treatments for the mentioned disease and precise details of their viral-based GT clinical trial studies in the last decade, then presents the advantages, disadvantages, and potential adverse events of GT. In conclusion, this review presents GT as a hopeful and growing field in healthcare that could offer cures to diseases that were previously thought to be untreatable.
Indexed as
Identifiers
What OpenQuestion holds
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 OpenQuestion graph.