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RNA and Nanotechnology Enable Wider Accessibility to Stem Cell Transplantation

Objective

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.

Coordinator

LUNDS UNIVERSITET
Net EU contribution
€ 150 000,00
Address
Paradisgatan 5c
22100 Lund
Sweden

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Region
Södra Sverige Sydsverige Skåne län
Activity type
Higher or Secondary Education Establishments
Other funding
No data