UNI-Large: precision gene editing techniques for congenital type 1A muscular dystrophy

Marc Güell Cargol

Universidad Pompeu Fabra, Spain

In 2017, we developed Uni-Large, a new generation of gene editing technologies that combines the precision of CRISPR with the effectiveness of vectors established in the clinic. We are currently developing the first treatment for congenital type 1A muscular dystrophy (MDC1A), although we expect that Uni-Large will be able to used in the treatment of other diseases.

MDC1A is a condition produced by mutations in LAMA2, a protein that exceeds the size limit of the adeno-associated virus (AAV) vectors, therefore, alternative therapies are needed. Uni-Large will provide a safe therapeutic opportunity.

It solves several challenges that impede a wider deployment of gene editing technologies, such as dependence on mutation and homologous recombination, and safety issues related to double-strand breaks and insertion mutagenesis.