Description du projet
Développer les cellules souches du sang du cordon ombilical à des fins de transplantation
Le sang du cordon ombilical est prélevé à la naissance et constitue une autre source de cellules souches hématopoïétiques (CSH). Cependant, le nombre de CSH du sang du cordon ombilical est insuffisant pour une transplantation clinique, ce qui entrave leur application clinique. Financé par le Conseil européen de la recherche, le projet RNable étudie la possibilité de développer ces cellules in vitro en amont de la transplantation. Les chercheurs ambitionnent d’acheminer des petits ARN dans les CSH en ayant recours à une méthodologie non toxique fondée sur la nanotechnologie qui maintient la fonction et la viabilité des CSH. Le projet permettrait d’améliorer les procédures de transplantation de CSH, pour le plus grand bien des milliers de personnes qui ne trouvent pas de donneurs admissibles.
Objectif
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.
Champ scientifique
Programme(s)
- HORIZON.1.1 - European Research Council (ERC) Main Programme
Régime de financement
HORIZON-AG-LS - HORIZON Lump Sum GrantInstitution d’accueil
22100 Lund
Suède