M34, Inc founded by Prof. Dr. Jude R. Samulski, widely regarded as the “father” of modern gene therapy which is a gene therapy company dedicated to advancing neuro regenerative treatments, have published a groundbreaking retrospective and prospective review on Adeno-Associated Viral (AAV) vectors in the prestigious journal Molecular Therapy.

Among its key highlights, the article for the first time introduces the innovative and pioneering use of AAV libraries in human decedents, a strategy spearheaded by M34, Inc. and known as the Human-to-Human (H2H) Project. Traditional AAV directed evolution strategy had incorporated in vitro cell, ex-vivo organs, rodent species and non-rodent species (such as pig, dog and no human primate). The traditional methods could not cross over the inherent genetic difference between species and complexities such ad epigenetic regulation, splicing mechanism and species/strain specific host factors. H2H approach represents a paradigm shift in vector discovery, with the potential to generate gene therapy tools precisely aligned with human biology.