Uni-large is a new generation of gene editing techniques combining the precision of CRISPR/cas9 and the efficiency of viral vectors established in clinic, which allows the insertion of multi-kilobase genome fragments.
Congenital muscular dystrophy Type 1A (MDC1A) is an early onset and life-threatening disease that affects 1-9 in 1,000,000 people worldwide. It is caused by mutations in the laminin alpha-2 gene, a large gene that exceeds the size limit of adeno-associuated viral vectors (AAV), used for in vivo gene therapy. Therefore, it is necessary to develop alternative therapies. Uni-large will provide a safe and therapeutic opportunity.
The advantages are the following:
- Safe and efficient, novel and specific treatment
- MDC1A is life-threatening, no treatment available
- Versatile strategy, applicable to other diseases
- Efficient for large genes