MSU Technologies

Recombinant Adeno-Associated Virus Capsid Mutants for Enhanced Transduction of Therapeutic Genetic Material in the CNS

Executive Summary

The largest limiting factor in viral-mediated gene therapy is the efficacy of gene delivery to the target cells, which is dependent on the level of transduction. This becomes particularly important for therapies directly injected into the brain or central nervous system (CNS) as only a limited volume can be injected and the procedure is highly invasive. MSU researchers have engineered recombinant adeno-associated virus (rAAV2) mutant capsids that are more efficient at transduction of cells of the CNS, allowing for a more effective therapy.

Description of Technology

rAAV is the leading method of gene therapy for the CNS. Many clinical trials, however, have showed low efficacy due to insufficient viral transduction of neurons. The rAAV mutants developed at MSU show a markedly heightened transduction efficacy in the rat striatum and hippocampus. This transduction is enhanced 3-fold over the non-mutated rAAV capsid. These capsid mutants could be highly beneficial for improving transduction and gene delivery in the CNS, therefore providing more potent and efficacious therapies for diseases such as Parkinson’s, Alzheimer’s, and Huntington’s.

Key Benefits

More efficient gene transfer: improve effectiveness and dosing requirements
More efficacious and safe gene therapeutics: reduces the amount of therapeutic material necessary, reducing potential immune response
Long lasting therapeutic effectiveness

Applications