基因微型启动因子将促进研究与治疗

University of British Columbia researchers have led the development of a new "toolbox of MiniPromoters" for research and future therapies on brain, spinal¹ cord（脊髓） and eye function. MiniPromoters are small segments（片段） of human DNA² with the ability to turn genes⁴ on and off at specific times and locations. They're important tools used by scientists and clinicians to mark cells, explore function and deliver therapeutic⁵ genetic⁶ medicine. Gene³ therapy, or targeted gene replacement⁷, is being investigated as potential therapy for neurodegenerative（神经变性的） disorders⁸ such as Alzheimer Disease, Parkinson Disease, Huntington Disease, Multiple Sclerosis（硬化） , Aniridia（无虹膜） and cancer.

Led by Elizabeth M. Simpson, UBC Medical Genetics Professor and Senior Scientist at the Centre for Molecular⁹ Medicine and Therapeutics at the Child & Family Research Institute, an international team of 64 researchers are making available 27 new MiniPromoters, greatly expanding the current limited pool of these vital tools for research and treatment of these disorders.

Using genome analysis, the research team identified new regions of human DNA that can activate¹⁰ a gene in certain brain regions but not others. Further research will focus on refining the gene expression patterns and optimizing¹¹ gene delivery methods in animal models.

Details of the project, called the Genome Canada Pleiades Promoter Project, are scheduled to be published online in PNAS Early Edition this week.