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Hemophilia A: an ideal disease to correct in utero.
Porada CD, Rodman C, Ignacio G, Atala A, Almeida-Porada G. Porada CD, et al. Front Pharmacol. 2014 Dec 11;5:276. doi: 10.3389/fphar.2014.00276. eCollection 2014. Front Pharmacol. 2014. PMID: 25566073 Free PMC article. Review.
There is thus a need for novel treatments, such as those using stem cells and/or gene therapy, which have the potential to mediate long-term correction or permanent cure following a single intervention. ...
There is thus a need for novel treatments, such as those using stem cells and/or gene therapy, which have the potential to mediate long-t …
Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene Delivery.
Porada CD, Almeida-Porada G. Porada CD, et al. J Genet Syndr Gene Ther. 2012 May 25;S1:011. doi: 10.4172/2157-7412.S1-011. J Genet Syndr Gene Ther. 2012. PMID: 23264887 Free PMC article.
There is thus a need for novel treatments that can promise long-term or permanent correction. In contrast to existing protein based therapeutics, gene therapy offers to provide a permanent cure following few, or even a single, treatment. ...
There is thus a need for novel treatments that can promise long-term or permanent correction. In contrast to existing protein based t …