Development of a nanoparticle-based gene editing technology for neurological applications

NIH RePORTER · NIH · UH3 · $499,996 · view on reporter.nih.gov ↗

Abstract

Abstract This supplement proposal focuses on investigating if CRISPR Cas9 ribonucleoproteins (RNPs) complexed to cell-penetrating peptides (CPPs) can rescue mice from Huntington’s disease, using the R6/2 mouse model. These experiments are based upon our recent studies demonstrating that CPPs can efficiently transfect CRISPR Cas9-RNPs neurons in the striatum after an intracranial injection using convection enhanced delivery (CED). We have selected the 2/6 mouse model for these studies because this model has recently been rescued with Cas9 based genome editing, using AAV delivered SaCas9. In this proposal we will build on our Preliminary Studies and will inject Cas9-RNP complexed to CPPs into the striatum and thalamus, via CED, and will investigate if we can edit the huntingtin (HTT) gene to rescue mice from Huntington’s disease. If successful, these experiments will identify the levels of genome editing needed in the brain to generate therapeutic outcomes in the Huntington’s animal model.

Key facts

NIH application ID
10619048
Project number
3UH3NS115599-04S1
Recipient
OHIO STATE UNIVERSITY
Principal Investigator
Krystof S Bankiewicz
Activity code
UH3
Funding institute
NIH
Fiscal year
2022
Award amount
$499,996
Award type
3
Project period
2019-09-15 → 2024-07-31