Descripción del proyecto
Expansión de las células madre de sangre del cordón umbilical para trasplantes
La sangre del cordón umbilical se recoge en el momento del parto y constituye una fuente alternativa de células madre hematopoyéticas (CMH). Sin embargo, el número de CMH de sangre del cordón umbilical es insuficiente para un trasplante clínico, lo cual impide su aplicación clínica. El equipo del proyecto RNable, financiado por el Consejo Europeo de Investigación, estudia la posibilidad de expandir estas células «in vitro» antes del trasplante. El objetivo de los investigadores es administrar pequeños ARN a las CMH mediante una metodología nanotecnológica no tóxica que mantiene la función y la viabilidad de las CMH. El proyecto podría mejorar los protocolos de trasplante de CMH y ayudar a miles de personas que no encuentran donantes aptos.
Objetivo
Every year, more than 50 000 patients receive blood stem cell transplantation as a curative treatment for diseases such as leukemia, immune deficiencies, and sickle cell anemia. For a successful transplantation, a matching donor who is willing to donate blood stem cells needs to be found. Worldwide, around 30% patients in need of a transplantation cannot find a suitable donor. Umbilical cord blood (UCB) is an easily obtainable alternative source of blood stem cells, but unfortunately most UCB units do not contain enough stem cells for a successful transplantation, which is why UCB is currently only rarely used in the clinic. Within the ERC project UNEXPECTED, we have identified several small RNA biomolecules that can be used to expand engraftable blood stem cells efficiently. However, it is difficult to safely deliver molecules to blood stem cells, and currently used methods are highly toxic. We recently applied cutting edge nanotechnology to solve this long-standing problem in stem cell biology. We established nanostraws as an efficient and gentle alternative delivery method with which both cell function and viability are fully maintained. Our method that allows efficient non-toxic delivery of blood stem cell-expanding RNAs will allow us to greatly increase the number of UCB units that can be used for transplantations. Our approach could provide a life-saving treatment option for the thousands of patients yearly with malignant or inherited diseases that currently are ineligible for a stem cell transplantation procedure.
Ámbito científico
Programa(s)
- HORIZON.1.1 - European Research Council (ERC) Main Programme
Régimen de financiación
HORIZON-AG-LS - HORIZON Lump Sum GrantInstitución de acogida
22100 Lund
Suecia