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Investigating Optimal Autologous Cellular Platforms for Prenatal or Perinatal Factor VIII Delivery to Treat Hemophilia A
- Source :
- Frontiers in Cell and Developmental Biology, Frontiers in Cell and Developmental Biology, Vol 9 (2021)
- Publication Year :
- 2021
- Publisher :
- Frontiers Media S.A., 2021.
-
Abstract
- Patients with the severe form of hemophilia A (HA) present with a severe phenotype, and can suffer from life-threatening, spontaneous hemorrhaging. While prophylactic FVIII infusions have revolutionized the clinical management of HA, this treatment is short-lived, expensive, and it is not available to many A patients worldwide. In the present study, we evaluated a panel of readily available cell types for their suitability as cellular vehicles to deliver long-lasting FVIII replacement following transduction with a retroviral vector encoding a B domain-deleted human F8 transgene. Given the immune hurdles that currently plague factor replacement therapy, we focused our investigation on cell types that we deemed to be most relevant to either prenatal or very early postnatal treatment and that could, ideally, be autologously derived. Our findings identify several promising candidates for use as cell-based FVIII delivery vehicles and lay the groundwork for future mechanistic studies to delineate bottlenecks to efficient production and secretion of FVIII following genetic-modification.
- Subjects :
- FVIII
Factor replacement
QH301-705.5
business.industry
Genetic enhancement
Transgene
mesenchymal stromal (stem) cell therapy
Cell Biology
Postnatal treatment
Bioinformatics
gene therapy
Viral vector
Cell and Developmental Biology
Immune system
endothelial progenitor cell
Severe phenotype
Perinatal factor
Medicine
hemophilia A
Biology (General)
business
Developmental Biology
Original Research
Subjects
Details
- Language :
- English
- ISSN :
- 2296634X
- Volume :
- 9
- Database :
- OpenAIRE
- Journal :
- Frontiers in Cell and Developmental Biology
- Accession number :
- edsair.doi.dedup.....00fedaf78e6772c4ee0d6baa5cf24a6b