Genetic engineering helps researchers overcome two major immune barriers to gene therapy

So far, there are two major immune barriers to treating Duchy muscular dystrophy with gene therapy: first, mutations in the antimyotrophic protein used to treat patients with ductum muscular dystrophy, which can turn into exogenetic proteins that trigger an immune response, and second, the adenovirus 6 (AAV-6) vector used to deliver antimyotrophic protein is a viral vector with inherent immunogenicity.
To overcome these obstacles, scientists from Stanford University and the University of Washington have recently developed a new technique to deliver engineered plasmid DNA to reduce the likelihood of an autoimmune response, according to research published recently in PNAS, entitled "Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of generapy of Duchenne dystrophy."
researchers say they have in fact applied the technique to humans: myelin, which can be used to deliver the treatment of multiple sclerosis, and insulin-based protein for the treatment of type 1 diabetes. In the latest study, researchers expanded the technology into gene therapy to reduce the immunogenicity of AAV vectors and the wild proteins produced by the disease that cause loss.
after recombining the AAV-6-tiny antimyostrophy protein gene through systematic injections to mdx/Mtrg2 mice, the researchers found that this traditional method did induce the body to produce antibodies that targeted antimyotrogen proteins and vectors. However, using an engineered DNA structure that encodes tiny antimyostrophy proteins can significantly reduce the body's immune response to antimyotrophy proteins and AAV-6 vectors in mice. The researchers found that the muscle strength of treated mice also improved significantly.
The data show that this method of coding disease-resistant proteins using engineered DNA protons has the advantage of avoiding immune side effects, thus overcoming key barriers to existing gene therapy and promoting unrestricted expression of functional genes for better functional therapeutic results. (Bio Valley)