ArticleThe Journal of clinical investigation2006
The leak stops here: platelets as delivery vehicles for coagulation factors.
Article in The Journal of clinical investigation, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 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
6 citing papers in PubMed, 12 citations in OpenAlex.
- Liposomal factor VIII as an efficient pharmaceutical system for the treatment of hemophilia.Iranian journal of basic medical sciences · 2024Article
- Doxorubicin-loaded platelets conjugated with anti-CD22 mAbs: a novel targeted delivery system for lymphoma treatment with cardiopulmonary avoidance.Oncotarget · 2017Article
- Embryonic stem cells as sources of donor-independent platelets.The Journal of clinical investigation · 2015Article
- Co-opting biology to deliver drugs.Biotechnology and bioengineering · 2014Review
- The use of PEGylated liposomes in the development of drug delivery applications for the treatment of hemophilia.International journal of nanomedicine · 2010Review
- Analysis of the spatial and temporal characteristics of platelet-delivered factor VIII-based clots.Blood · 2008Article
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Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy is an attractive approach for the treatment of hemophilia, as continuous expression of donated clotting factor VIII (FVIII) DNA would ensure clotting factor replacement at constant circulating levels rather than at the peaks and troughs that characterize the current protein infusion therapeutic approach. In this issue of the JCI, Shi et al. describe an interesting variant of a gene transfer approach for hemophilia (see the related article beginning on page 1974). They show that targeted expression of FVIII in megakaryocytes, with storage in the alpha-granules of platelets, has the advantage of delivering clotting factors directly to the site of an injury, where platelets accumulate in large numbers and undergo activation accompanied by release of granule contents. Earlier clinical experience with gene transfer into hematopoietic cells highlighted the potential safety risks of this approach, but an F8 transgene may represent a lower risk than transgenes for growth factors or their receptors.
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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.