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PRLR-CRISPR-Cas9
- Development stage
- Preclinical
- Lead developer
- University of Arizona
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Intranasal
01
Overview
PRLR-CRISPR-Cas9 is an experimental CRISPR-based gene therapy designed to selectively delete or knock down the prolactin receptor (PRLR) gene. In preclinical research focused on migraine, this agent is administered intranasally to target the trigeminal ganglia, effectively reducing the expression of both long and short PRLR isoforms. This disruption of prolactin signaling prevents the sensitization of trigeminal nociceptors that typically occurs in response to stress-induced prolactin release. Because this mechanism is specifically linked to female-selective migraine-like pain, PRLR-CRISPR-Cas9 represents a potential therapeutic strategy for addressing the female prevalence of migraine and other stress-related neurological disorders.
- Other names
- PRLR-targeting CRISPR-Cas9nasal PRLR-CRISPR-Cas9
02
Targets
PRLR (Prolactin receptor)
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