Effects Of Premature Truncation Of The UBE3A Antisense transcript On UBE3A Imprinted Expression
This grant continued ASF funding to one of the premier AS scientists, Dr. Joe Wagstaff, and his laboratory. The aim of this project was a bold attempt to create therapeutic effects in the mouse model by genetically engineering a control element of UBE3A expression. This element was modified by advanced methods of genetic engineering and embryonic cell treatment. During the course of the study Dr. Wagstaff unfortunately died and this project was discontinued due to the closure of his laboratory and the funds for it returned to the foundation. Dr. Wagstaff was subsequently honored by ASF as an outstanding investigator and ASF now provides grant funding opportunities to young science investigators through the Wagstaff Fellowship program.