ArticleCell stem cell2013
Broadening the indications for hematopoietic stem cell genetic therapies.
Article in Cell stem cell, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 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
9 citing papers in PubMed.
- Human Lentiviral Gene Therapy Restores the Cellular Phenotype of Autosomal Recessive Complete IFN-γR1 Deficiency.Molecular therapy. Methods & clinical development · 2020Article
- Getting the Most: Enhancing Efficacy by Promoting Erythropoiesis and Thrombopoiesis after Gene Therapy in Mice with Hurler Syndrome.Molecular therapy. Methods & clinical development · 2018Article
- Gene therapy research in Asia.Gene therapy · 2017Review
- Evolving Gene Therapy in Primary Immunodeficiency.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Review
- Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.The Journal of clinical investigation · 2016Article
- In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases.Cell stem cell · 2016Article
- Unexpected help: mTOR meets lentiviral vectors.Blood · 2014Article
- Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases.Stem cells translational medicine · 2014Review
- Platelets are efficient and protective depots for storage, distribution, and delivery of lysosomal enzyme in mice with Hurler syndrome.Proceedings of the National Academy of Sciences of the United States of America · 2014Article
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Authors and funding
1 author.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The use of recombinant retroviral vectors to effect corrective genetic therapies in hematopoietic stem cells (HSCs) has long been predicted to revolutionize medicine. Two recent papers in Science now show that this technology could be considered as effective as, and perhaps superior to, allogeneic HSC transplants in some rare diseases.
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Identifiers
24012366What 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.