VHH Gene Therapy (small size)

Harnessing VHHs in Gene Therapy: Expanding Therapeutic Reach in the Cytoplasm

Due to their small size (~120 amino acids), the cDNA encoding a VHH is barely 0.35-0.4 kb. This makes them adaptable to gene therapy and indeed a VHH is easily accommodated within the genome of an AAV or of a lentivirus. The combination between such a gene therapy virus and a VHH unlocks the entire cytoplasm, as well as subcellular organelles, as a therapeutic playground, where numerous targets reside that were previously almost impossible to reach.