A new technology that delivers two halves of a gene separately could enable gene therapy treatments of muscular dystrophies.

The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size. The ...

With over 100 publications in international journals, his groundbreaking work has earned him ... In 2014, he founded VectorBuilder, a revolutionary online platform for vector design and ordering that ...