Originally published on August 22, 2013.
The grant award to Dr. Yimin is the fourth drug discovery project funded by Cure SMA in 2013 with a total investment of $550,000. This program will systematically assess the effect of backbone chemistry on the therapeutic efficacy of Antisense Oligonucleotides (ASO) that target the ISS-N1 region of the SMN2 RNA. ASOs that bind to the ISS-N1 region will be compared. This is the binding region of the drug ISIS-SMNRx that is currently being tested by Isis Pharmaceuticals in SMA clinical trials. This funding is being awarded to Dr. Yimin Hua in the laboratory of Dr. Adrian Krainer at Cold Spring Harbor Laboratory. Both scientists are long-term collaborators with Isis Pharmaceuticals and actively participated in the pre-clinical development and characterization of the mechanism of action of ISIS-SMNRx.
Meet Dr. Hua:
Who are you?
I am a molecular biologist, and currently a Research Investigator at the Cold Spring Harbor Laboratory (CSHL). I obtained my Ph.D. degree in molecular endocrinology in 1998 from Sun Yat-sen University in China. During 2000-2004, I worked on SMA pathogenesis with Professor Elliot Androphy and Dr. Jianhua Zhou in the Tufts Medical Center and University of Massachusetts Medical School. In 2004, I joined Professor Adrian Krainer at the CSHL to develop antisense drugs to treat SMA. The area of my expertise is focused on studying gene expression regulation, particularly RNA splicing regulation, and using antisense strategies to design therapeutic approaches .
How did you first become involved with SMA research?
In May of 2000, I came to the USA as a postdoctoral fellow in Elliot Androphy’s lab. I was shocked to learn that the lack of SMN causes SMA, the devastating infantile disease. I immediately felt very much interested in studying the pathogenesis of the disease and developing therapeutic drugs.
What is your current role in SMA research?
I have been working on SMA since I moved to the USA in 2000. In the past few years I focused on developing antisense drugs to treat SMA through correcting SMN2 splicing. The antisense drug candidate is now in the Clinical Phase II trials. I also recently uncovered that peripheral tissues play essential roles in rescuing SMA mouse models. My research effort is currently focused on optimizing antisense therapy for SMA and searching for SMN downstream gene targets that are critical in SMA pathology.