full-length human dystrophin plasmid

Full-length human dystrophin plasmid is a gene therapy candidate designed for the treatment of Duchenne muscular dystrophy (DMD). Developed by EndoFold in collaboration with Ansa Biotechnologies, the therapy utilizes a large DNA plasmid (~23 kb) containing a codon-optimized ~11.5 kb expression cassette for the complete human dystrophin gene. This approach aims to overcome the limitations of traditional adeno-associated virus (AAV) vectors, which are restricted by a ~4.7 kb packaging capacity and thus require the use of truncated "micro-dystrophin" constructs. By delivering the full-length gene, the therapy seeks to restore the expression of the entire functional protein. Preclinical proof-of-concept studies have demonstrated successful synthesis and high-fidelity expression of the full-length protein in human HEK293T cells.

Other names

full-length human dystrophin construct