Gene therapy breakthrough
We're delighted to share some important research news with you. Last year we announced a partnership with Solid GT, a subsidiary of Solid Biosciences, devoted to developing a gene therapy treatment for DMD.
Earlier this month, one of the labs working with Solid GT published some exciting data, showing that they have successfully and safely transferred a mini version of the dystrophin gene to the muscles of dogs, using a safe virus called AAV. The virus successfully carried the mini gene to the skeletal muscle, and the heart and diaphragm.
This is an important step on the road to clinical trials in humans.
The study was carried out at Missouri University. The team leader, Professor Dongsheng Duan, said:“This discovery took our research team more than 10 years, but we believe we are on the cusp of having a treatment for the disease”
We’d like to congratulate Professor Duan and his team. Professor Duan is a valued member of the SOLID GT Scientific Advisory Board and we are delighted to have partnered with them to work in this exciting field. Read more here.
We never forget that our aim is to #EndDuchennein10 And we are working every day to get there
Published on 3 November 2016Share this article Categories Uncategorised