RT Journal Article
T1 Treatment of congenital coagulopathies, from biologic to biotechnological drugs: the relevance of gene editing (CRISPR/Cas)
A1 De Pablo Moreno, Juan Andrés
A1 Miguel Batuecas, Andrea
A1 Rodríguez Merchán, E. Carlos
A1 Liras Martín, Antonio
AB Congenital coagulopathies have, throughout the history of medicine, been a focus of scientific study and of great interest as they constitute an alteration of one of the most important and conserved pathways of evolution. The first therapeutic strategies developed to address them were aimed at restoring the blood components lost during hemorrhage by administering whole blood or plasma. Later on, the use of cryoprecipitates was a significant breakthrough as it made it possible to decrease the volumes of blood infused. In the 1970′ and 80′, clotting factor concentrates became the treatment and, from the 1990's to the present day, recombinant factors –with increasingly longer half-lives– have taken over as the treatment of choice for certain coagulopathies in a seamless yet momentous transition from biological to biotechnological drugs. The beginning of this century, however, saw the emergence of new advanced (gene and cell) treatments, which are currently transforming the therapeutic landscape. The possibility to use cells and viruses as well as specific or bispecific antibodies as medicines is likely to spark a revolution in the world of pharmacology where therapies will be individualized and have long-term effects. Specifically, attention is nowadays focused on the development of gene editing strategies, chiefly those based on CRISPR/Cas technology. Rare coagulopathies such as hemophilia A and B, or even ultra-rare ones such as factor V deficiency, could be among those deriving the greatest benefit from these new developments.
PB Elsevier
SN 0049-3848
YR 2023
FD 2023-10-14
LK https://hdl.handle.net/20.500.14352/103600
UL https://hdl.handle.net/20.500.14352/103600
LA eng
NO 2023 Acuerdos Transformativos CRUE
NO Asociación para la Investigación y Cura del Déficit de Factor V
NO Universidad Complutense de Madrid
NO Banco Santander
DS Docta Complutense
RD 11 abr 2025