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AAV-ERCC5 gene therapy
- Development stage
- Preclinical
- Lead developer
- University of Minnesota
- Modality
- Gene Therapies
- Administration
- Intravenous, Intrathecal
01
Overview
AAV-ERCC5 gene therapy is an investigational adeno-associated virus (AAV)-based gene replacement therapy designed to treat Xeroderma pigmentosum-cockayne syndrome (XP-CS) caused by mutations in the *ERCC5* (XPG) gene. XP-CS is a severe DNA repair disorder characterized by neurodegeneration, premature aging, and photosensitivity. The therapy aims to deliver a functional copy of the *ERCC5* gene to restore nucleotide excision repair (NER) activity. Preclinical studies are evaluating various AAV capsids (such as AAV9 and AAVDJ) and promoters (Ef1α and CP040) via intravenous or intrathecal administration to optimize vector uptake and protein expression in the central nervous system and other tissues.
- Other names
- ERCC5 gene therapyERCC-5 gene therapyERCC 5 gene therapyXPG gene therapy
02
Targets
DDR (DNA damage response)Excision repair cross-complementation group 5 – Breast cancer type 1 susceptibility protein interactionPCNA (Proliferating cell nuclear antigen)ERCC5–XPA (Excision repair cross-complementing rodent repair deficiency, complementation group 5 – Xeroderma pigmentosum, complementation group A interaction)ERCC5-RPA (Excision repair cross-complementation group 5 – Replication protein A complex)
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