ReviewBrain & development2025
Hematopoietic stem cell gene therapy of neurometabolic lysosomal storage diseases.
Review in Brain & development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
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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
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
3 citing papers in PubMed.
- Biologic Therapies for Alleviating Neurodegeneration in Lysosomal Storage Diseases.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026Review
- GAA engineered to improve cellular uptake enhances correction in a preclinical hematopoietic stem cell gene therapy model of Pompe disease.Molecular therapy. Advances · 2026Article
- Updated EBMT/ESID inborn errors working party guidelines for haematopoietic stem cell transplantation for inborn errors of immunity and metabolism.Bone marrow transplantation · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Neurometabolic disorders are rare, inherited monogenic diseases arising from mutations in genes whose products are essential for brain functions and cause local accumulation of toxic substrates. Central nervous system involvement can be severe, is progressive and frequently appears early in life. Current treatment options for neurometabolic disorders are represented mainly by enzyme replacement therapy (ERT) and allogeneic hematopoietic stem cell transplantation (HSCT) which do not sufficiently address clinical manifestations and leave patients with a substantial residual disease burden. Given this unmet medical need, alternative strategies based on genetic manipulation of patient's cells have been developed. Hematopoietic stem progenitor cells-gene therapy (HSPC-GT) entails the harvest autologous HSPCs which are ex-vivo manipulated by means of viral vectors to express the therapeutic gene and infused back into the patient after chemotherapy-based preparation. Modified HSPCs engraft and differentiate into the various hematopoietic cell lineages, producing the functional enzyme at either normal or supranormal levels. The number of clinical trials with HSPC-GT in neurometabolic disorders is rapidly increasing and some HSPC-GT products have recently received market approval. This review focuses on HSPC-GT strategies summarizing the most recent developments in the field of neurometabolic disorders.
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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 OpenQuestion graph.