Gene Therapy for CMT1BCurrently, no cure exists for Charcot-Marie tooth type 1B (CMT1B). Inventors and specialists in Gene Therapy at Nationwide Children’s Hospital invented a methodology along with sequences for using microRNAs (miRNA) to inhibit and replace abnormal expressions of the myelin protein zero (MPZ) gene. Affecting 1 in 30,000 people, CMT1B is caused by more than 200 mutations of the MPZ, the essential protein needed for a healthy and efficient peripheral nervous system. The accumulation of mutant MPZ genes will result in, but not limited to, muscle weakness, atrophy, lost of sensation in the lower legs and feet and sensory loss. These methods can treat, delay the progress of and prevent diseases caused by the mutations. This gene therapy knocks down MPZ gene expression with nucleic acid encoded artificial microRNAs hybridized to target nucleic acid sequences at the mRNA level and a nucleic acid encoding a codon-optimized MPZ gene resistant to the artificial miRNA (resMPZ). Then, using adeno-associated viruses (AAVs) to package and deliver miRNA and resMPZ into Schwann Cells (SCs) via injection. As a result, mutated MPZ expression in SCs are inhibited and healthy levels of the normal MPZ protein are maintained. |
Tech ID2021-026 College(None) Licensing ManagerInventorsCategories(None) |
