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CRISPR-based genome editing (nonviral, SNCA transcriptional modulation)
- Development stage
- Preclinical
- Lead developer
- Innovative Genomics Institute
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Parenteral
01
Overview
CRISPR-based genome editing (nonviral, SNCA transcriptional modulation) is an experimental, nonviral gene therapy designed for the permanent and precise downregulation of alpha-synuclein (αSyn) to treat Parkinson's disease and other alpha-synucleinopathies. Developed by researchers at the University of California, Berkeley, the Innovative Genomics Institute, and its spinout Editpep, this approach utilizes a CRISPR-based system (such as the peptide-mediated Neuro-PERC platform) to target and perturb specific regulatory motifs within the endogenous SNCA locus. By modulating transcription rather than completely ablating the gene, the therapy aims to achieve controlled, moderate suppression of αSyn expression, thereby reducing pathological burden while preserving essential physiological functions of the protein.
- Other names
- Neuro-PERC
02
Targets
SNCA (Alpha-synuclein)
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