ReviewMolecular therapy : the journal of the American Society of Gene Therapy2025
Clinical hematopoietic stem cell-based gene therapy.
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 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
17 citing papers in PubMed.
- Single-cell vector copy number analysis of phenotypically defined long-term hematopoietic stem cells for gene therapy safety assessment.Molecular therapy. Advances · 2026Article
- Defining global priorities in hematopoietic transplantation and cellular therapy: a statement paper from EBMT Global Committee.Bone marrow transplantation · 2026Article
- Engineered lipid nanoparticles for in vivo and durable editing of haematopoietic stem cells within humanized mice.Nature biomedical engineering · 2026Article
- Genetic influences on haematopoiesis.Nature reviews. Genetics · 2026Review
- CD117 epitope-shielded hematopoietic stem cell transplantation with toxin-free conditioning and in vivo selection ameliorates a β-thalassemia model.bioRxiv : the preprint server for biology · 2026Article
- Flow-Induced Yap/Taz Signaling Balances Endothelial and Hematopoietic Stem Cell Fates.bioRxiv : the preprint server for biology · 2026Article
- Molecular and therapeutic frontiers in anemia therapy.The Journal of clinical investigation · 2026Review
- Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026Review
- Engraftment Outcome of CRISPR/Cas9-Edited Hematopoietic Stem Cells for Genetic Diseases: A Systematic Review and Meta-Analysis of Preclinical Evidence.Journal of hematology · 2026Article
- Epigenome editing of human hematopoietic stem cells enables sustained and reversible thrombosis prevention.bioRxiv : the preprint server for biology · 2026Article
- Genome editing between wonder and rejection.Frontiers in bioengineering and biotechnology · 2026Article
- Strategies for the Prevention and Control of Complications of Pathological Myopia: A Systematic Review of Research Progress from Axial Control to Target Organ Protection.Clinical ophthalmology (Auckland, N.Z.) · 2026Review
- Synthetic biology and genome engineering in support of human, agricultural, and environmental security.Frontiers in bioengineering and biotechnology · 2026Article
- Cell-drug conjugates: a novel drug delivery system for cancer therapy.Theranostics · 2026Review
- Gene Therapy of Beta Hemoglobinopathies.Biomedicines · 2025Review
- Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- CRISPR/Cas-edited iPSCs and mesenchymal stem cells: a concise review of their potential in thalassemia therapy.Frontiers in cell and developmental biology · 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
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hematopoietic stem cell (HSC)-based gene therapies have seen extraordinary progress since their initial conception, now fundamentally transforming the treatment paradigms for various inherited hematologic, immunologic, and metabolic conditions-with additional use cases under exploration. Decades worth of work with advances in viral vector technologies and cell manufacturing have paved the way for HSC gene therapy with marked improvement in the safety and efficiency of gene delivery into HSCs. These have been augmented by the recent rise of innovative genome-editing techniques, particularly using clustered regularly interspaced short palindromic repeats CRISPR-associated proteins (CRISPR-Cas)-based technologies, which have enabled more precise and reproducible genome alterations in HSCs and fostered opportunities for targeted gene modification or gene correction. These breakthroughs have led to the development of many active clinical trials and culminated in the recent federal regulatory-agency approvals of multiple clinical HSC gene therapies for various indications that are now becoming available across different geographies. These treatments aim to offer significant, long-lasting benefits to patients worldwide without the toxicities of alternative treatment approaches. This review explores the history and advancements in HSC gene therapies and provides a comprehensive overview of the latest clinical innovations and cell-therapy products. Further, it concludes with a discussion of the persistent challenges that have limited adoption and potential future opportunities that aspire to enable curative treatment of many different patients through such personalized medicines.
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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.