Development of an effective gene replacement therapy for NF1 is complicated by the large size of the gene affected in this disease, preventing its packaging into the safe DNA carriers used in therapeutic gene delivery for other genetic diseases. The overall aim of this proposal is to therefore develop a gene replacement therapy that has applicability to all NF1 patients, using a novel DNA carrier for the targeted delivery of the complete coding sequence of the NF1 gene. Efficient delivery, expression and function of neurofibromin will then be examined. Translational development of the optimized carrier and NF1 DNA will be made through treatment and analysis of two mouse models of the disease.
Investigators
Deeann Wallis, PhD
University of Alabama, Birmingham
Linda Popplewell, PhD
Royal Holloway University of London
Deeann Wallis, PhD
University of Alabama, Birmingham
Linda Popplewell, PhD
Royal Holloway University of London