Beta-amyloid plaque elimination throughout the brain with a gene therapy vector allowing for widespread therapeutic protein dissemination
A limitation of viral‑mediated gene therapy for neurological diseases is the necessity for a focal injection of virus with limited ability to transduce cells across the entire brain away from the focal point of injection. What is needed is a way for the viral expressed therapeutic protein produced by transduced cells to disseminate away from the injection site to effect cells across the entire span of the brain.
This technology is a gene therapy vector and method to deliver a therapeutic protein to the brain of neurological disease patients (i.e. Alzheimer’s patients) which in our experimental models has been demonstrated to virtually eliminate beta-amyloid. The main advantage of this vector, is that it is adenovirus-associated virus based, which is known to be safe, and the therapeutic protein it expresses is modified for constitutive transfer into the nucleus (no post-processing required) and it is also a secretary protein fused with a cell penetrating peptide to allow for diffusion of the therapeutic protein across the brain and uptake across the cell membrane and into the nucleus of adjacent cells. The end result is widespread dissemination of the therapeutic protein from the transduced cells throughout the brain where it can reach its target (i.e. the nucleus) and demonstrate therapeutic effect in non‑transduced cells.
- AAV’s have a proven safety profile.
- The vector system allows for widespread dissemination of the therapeutic protein throughout the brain beyond the injection site or point of entry sites of localized transduction.
- Preliaminary pre‑clinical results show virtually complete elimination of beta amyloid plaques (and tau).
- The vector system and method is applicable to any gene therapy for neurological disease.
- Alzheimer’s disease.
- Parkinson’s disease.
- Neurodegenerative disease.
- Any gene therapy application in the brain.
Lawson Health Research Institute are actively seeking a development/commercialization partnership.
- Development partner
- Commercial partner