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AAV8-SpCas9-PAH transgene
- Development stage
- Preclinical
- Lead developer
- Oregon Health & Science University
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
AAV8-SpCas9-PAH transgene is a descriptive name for an experimental dual-vector, liver-directed **AAV8 CRISPR gene-editing intervention** evaluated in neonatal murine phenylalanine hydroxylase deficiency. One AAV8 vector delivers *Streptococcus pyogenes* Cas9 and a guide targeting the *Cypor* locus, while the companion AAV8 donor vector provides a phenylalanine hydroxylase transgene repair template for homology-directed insertion. The intended therapeutic effect is restoration of hepatic PAH activity to lower phenylalanine concentrations in phenylketonuria; in the supplied CYPOR-targeting study, initial phenylalanine lowering was not maintained during acetaminophen-mediated hepatocyte selection. Related OHSU work has shown that pharmacologic inhibition of competing DNA-repair pathways can improve PAH-transgene insertion and reduce serum phenylalanine in murine PKU models. ([biorxiv.org](https://www.biorxiv.org/content/10.1101/2025.02.24.639678v1))
- Other names
- AAV8-SpCas9-PAH transgeneAAV-8-SpCas9-PAH transgeneAAV 8-SpCas9-PAH transgene
02
Targets
PAH (Phenylalanine hydroxylase)
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