Protein trans-splicing: optimization of intein-mediated GFP assembly as a model for the development of gene therapy.

ABSTRACT: Adeno-associated virus (AAV)-based gene therapy has become one of the key directions of modern translational medicine geared towards treatment of hereditary disorders by means of gene replacement. At the moment, about 5,000 different syndromes are associated with mutations in large genes, which presents a great problem due to the AAV packaging capacity of 5 kilobases. The main strategies for overcoming this obstacle were the creation of truncated gene versions, overloading the viral vector, and separate delivery of partial genetic material to restore the whole gene at the level of DNA, RNA, or protein. At present, genome editing via prime editors, most effectively delivered by AAV, relies on the intein pair used to restore the protein complex. The amazing integration speed of intein-based

SUBMITTER: Brovin A 

PROVIDER: S-EPMC11614648 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature