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AAV-mediated adenine base editor
- Development stage
- Preclinical
- Lead developer
- Tianjin Medical University Cancer Institute and Hospital
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Injection
01
Overview
AAV-mediated adenine base editor is a gene therapy platform that utilizes adeno-associated virus (AAV) vectors to deliver adenine base editing (ABE) components for precise genomic modification. In the context of non-small cell lung cancer (NSCLC), a specific high-fidelity variant known as A8E-N108Q-R26G (spCas9-A8EQR) has been developed to target the STK11 Q37* (c.109C>T) nonsense mutation. This dual-AAV system enables the conversion of the mutant adenine back to guanine, thereby restoring the expression of the tumor suppressor LKB1. The restoration of LKB1 activity reactivates downstream signaling that stabilizes BACH1 and suppresses NRF2, leading to increased reactive oxygen species (ROS) levels and enhanced radiosensitivity in tumor cells. This approach represents a targeted strategy to overcome radiation resistance driven by STK11 mutations.
- Other names
- AAV-mediated ABE delivery
02
Targets
Genomic DNA locus specified by the sgRNA protospacer and PAM
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