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Experiences with mRNA

from life-science researchers

supported by the mRNA experts at RiboPro





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Elena Levtchenko - KU Leuven, Belgium

"Our group (Cystinosis Research Group, KU Leuven, Belgium) consisting of clinician-scientists (Elena Levtchenko, Koenraad Veys), molecular biologists (Bert van den Heuvel, Fanny Arcolino), viral vector technology engineer (Rik Gijsbers) and PhD student (Tjessa Bondue) studies inherited metabolic diseases cystinosis. This devastating disorder is caused by cystine accumulation in all cells of the body, but primary affects kidneys leading to end stage renal failure requiring dialysis and transplantation. Later in life, cystinosis also affects other organs such as eyes, thyroid, neural system and muscles severely affecting patients’ quality of life and causing early mortality. The only available drug for cystinosis an amino thiol cysteamine depleting cells from cystine only postpones disease progression, but offers no cure. Aiming at finding curative treatment, we have established collaboration with prof. Roland Brock from Radboud UMC and Mercurna/RiboPro to investigate mRNA-based therapy for cystinosis. High quality, stable and non-immunogenic mRNA generated by Ribopro is extremely suitable to meet our needs. The company also offers the qualified expert advice allowing the optimization of our experimental protocols. Our preliminary data in vitro cell model of cystinosis are very promising as we observe the restauration of the functional cellular phenotype. A joint funding by Health Holland Kickstarter grant will allow our research group together with Mercurna to apply RNA-based therapy in animal models of cystinosis (zebrafish and rat) to generate proof-of-principle data for the clinical studies. This pioneering work will be fundamental for a broad spectrum of genetic kidney disorders for which no curative therapies are available."

Aviad Pato - Gamida Cell, Israel

"At Gamida Cell, our goal is to develop a new generation of therapeutics for liquid and solid cancers and serious blood diseases using advance cell therapy. To achieve this goal, we require high quality, ultra-pure mRNA to maximize target protein expression in our primary NK cells. RiboPro provided both the expert support and synthesized the high-quality mRNA to make our first gene manipulation experiments a resounding success. Furthermore, RiboPro was able to provide the mRNA constructs at an inordinate speed thus matching our companies fast-pace environment and the constructs always proved to be reliable and of the highest standard. Now we are happy to say that the RiboPro is mRNA partner of choice."

Jitske Jansen - Radboudumc, the Netherlands

"My research aim is to model glomerular disorders in human iPSC-derived kidney organoids. To help achieve this goal, I use mRNA from RiboPro to induce expression of certain proteins of interest in these 3D cell cultures. Initially my results with mRNA were suboptimal, however with the support and dedication from the professionals at RiboPro, I was able to further optimize my experiments in such a way that my experiments turned out more successful.”