The Molecular Neurobiology Research laboratory is focused on developing and applying advanced gene-editing technologies for the treatment of Rett syndrome.
Classical gene therapy involves replacing a defective gene with a healthy one. This is most commonly done by removing cells from the body, using genetic engineering techniques to target and change the defective sequences in the DNA, and then reinserting the cells into the body.
When working with brain disorders, however, this approach is not viable due to the complex architecture of the brain and the inability of brain cells to divide. Thus it is not possible to remove brain cells, correct their defective sequences and place them back in the brain.
Using innovative gene-editing tools such as “CRISPR-Cas9”, the Molecular Neurobiology Research team are exploring an avenue of gene therapy beyond this conventional approach. This involves developing an intravenous therapy incorporating CRISPR-Cas9 that can cross the blood-brain-barrier, the brain’s gate-keeper, and specifically target the mutated MECP2 gene in affected cells. Replacing the disease-causing mutation with a normal copy of the gene would result in a permanently edited copy of the gene, and the potential to provide a life-long cure for patients with Rett syndrome.