Current Research Focus “Drug Repurposing”
Research4ml aims to support and accelerate research that could lead to finding a cure for ML or slowing down the progression of the disease. Existing research supports the idea that a combination of modes of therapy is necessary to address all affected tissues in ML, for instance gene therapy in combination with certain drugs/compounds.
Considering the limited lifespan of patients with ML a drug repurposing approach could offer a solution in a relatively shorter amount of time. Drug repurposing aims to identify a novel clinical use for an existing drug approved for a different indication [1].
Research Project “ML2TREAT”
Research4ml raises funds to support the research project “ML2TREAT” by two highly experienced experts in lysosomal storage diseases: Prof. Thomas Braulke (UKE Hamburg, Germany) and Prof. Diego Medina (TIGEM Neapel, Italy). Prof. Braulke in specific has significant experience into investigating ML II.
Thanks to generous support from the Yash Ghandi Foundation, initial funding for the research project is already secured. With your donation, we can help ensure that the project can continue to be financed.
Prof. Dr. Thomas Braulke has been a professor of biochemistry at the University Hospital Hamburg Eppendorf (UKE) since 1999. He has been researching mucolipidosis type 2 for many years and has published numerous valuable results on ML 2. For instance it was it was Dr. Stefan Tiede from Prof. Braulkes department who identified the gene responsible for ML 2 and ML 3, so that a clear diagnosis and prenatal diagnosis was made possible. To our knowledge, there is no medical scientist who has done more research on ML 2 than Thomas Braulke.
Prof. Diego Medina is Head of the High Content Screening Facility at the Telethon Institute of Genetics and Medicine (TIGEM). Since April 2019, he has also held a position at the University of Naples “Federico II” as Associate Professor in Biology at the Medical Genetics Unit, Department of Medical and Translational Science. Prof. Medina’s research group focuses on the study of the fundamental biological role of lysosomal function in health and disease using state-of-the-art high content imaging (HCI), cell biology, and OMICs approaches.
References
[1] Jourdan, J. P., Bureau, R., Rochais, C., & Dallemagne, P. (2020). Drug repositioning: a brief overview. Journal of Pharmacy and Pharmacology, 72(9), 1145-1151.