A shot in the bone corrects a genetic disease
- PMID: 25849425
- PMCID: PMC4395793
- DOI: 10.1038/mt.2015.38
A shot in the bone corrects a genetic disease
Comment on
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Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A.Mol Ther. 2015 Apr;23(4):617-26. doi: 10.1038/mt.2015.20. Epub 2015 Feb 6. Mol Ther. 2015. PMID: 25655313 Free PMC article.
References
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- Chuah MK, Evens H., and, VandenDriessche T. Gene therapy for hemophilia. J Thromb Haemost. 2013;11 suppl. 1:99–110. - PubMed
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- Moayeri M, Ramezani A, Morgan RA, Hawley TS., and, Hawley RG. Sustained phenotypic correction of hemophilia A mice following oncoretroviral-mediated expression of a bioengineered human factor VIII gene in long-term hematopoietic repopulating cells. Mol Ther. 2004;10:892–902. - PubMed
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