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[Gene therapy for hemophilia: feasible in principle but not yet clinically applicable]

Autor: Mm, Eb, Fh, Schagen, van Ormondt H, Rob Hoeben

Publikováno v: Europe PubMed Central

The current treatment of haemophiliacs consists of injection of concentrates of blood clotting factors VIII (haemophilia A) and IX (haemophilia B). The inconvenience of the multiple injections needed, and the risk of transmission of infectious agents

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::9d6c06001b33f04ce7c99cb52b8c4fdc
https://pubmed.ncbi.nlm.nih.gov/9623157

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Factors impeding efficient expression of factor VIII complementary DNA minigenes

Autor: Rob Hoeben, Fj, Fallaux, Fh, Schagen, Mm, Eb, van Ormondt H, van der Eb A

Publikováno v: Europe PubMed Central

Haemophilia A is a bleeding disorder that affects approximately 1 in 10,000 males. It is caused by a deficiency of functional blood-clotting factor VIII. Protein-replacement therapy has been effective as treatment, resulting in a vast improvement in

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::1dd13779426cf20843ca72e4c1ea6c76
http://europepmc.org/abstract/med/9607109

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