We have had the privilege to bring two renowned scientists on board who will lead Hope for Luka-funded pre-clinical research studies: Dr. Dongsheng Duan of the University of Missouri and Dr. Annemieke Aartsma-Rus of the Department of Human Genetics of the Leiden University Medical Center.
Dr. Duan and Dr. Aartsma-Rus are considered to be two of the best research scientists in the field of genetics in Muscular Dystrophy.
Our goal is to implement this blueprint for Lukian Keyghobadi, the six-year-old son of Hope for Luka founder Mazi Keyghobadias and then repeat it for other patients.
We are currently focused on funding and starting Project "Gray Area".This project is focused on creating a blueprint for preclinical studies for patients with rare deletions, duplications or nonsense mutations.
The first patient of the Project "Gray Area" is Lukian Keyghobadi. The research aims to study the use of lncRNA to preserve dystrophin and to create a mice model of the genetic deletion/duplication to test the research's findings in an in-vivo setting.
Dr. Duan's team at the University of Missouri will collaborate with Dr. Aartisma-Rus of Leiden University Medical Center in Netherlands on Project "Gray Area" as follow:
- Dr. Aartsma-Rus will perform research through Leiden University on the use of lncRNA to preserve dystrophin protein for Luka's deletion. The lncRNA study takes one year to complete, and will benefit exon-skipping studies, rare brain disease research as well as cancer research
- Dr. Duan and his team will produce a mice model for Luka's genetic deletion through University of Missouri.
Cost of study
Funds raised for the studies will be provided to University of Missouri and Leiden University in the form of donation as follow:
- Dr. Aartsma-Rus: $80,000
- Dr. Duan: $50,000
Targeted Start Dates
- Dr. Aartsma-Rus: 09/01/2023
- Dr. Duan: 09/01/2023
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