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.