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AAV2-FPC-CTD
- Development stage
- Preclinical
- Lead developer
- Mayo Clinic
- Modality
- Gene Therapies
01
Overview
AAV2-FPC-CTD is an adeno-associated virus serotype 2 (AAV2) gene therapy being developed by researchers at the Mayo Clinic for the treatment of autosomal recessive polycystic kidney disease (ARPKD). ARPKD is caused by mutations in the *PKHD1* gene, which encodes fibrocystin (FPC). Because the full-length *PKHD1* gene is too large for standard AAV packaging, AAV2-FPC-CTD delivers a transgene encoding the C-terminal domain of fibrocystin (FPC-CTD). This fragment translocates to the nucleus, where it suppresses cystogenesis and reverses profibrotic tubular phenotypes. In preclinical studies using human pluripotent stem cell-derived kidney organoids, AAV2-FPC-CTD demonstrated strong tubular tropism, rescued primary cilia, and prevented tubular DNA damage, senescence, and cyst formation.
- Other names
- AAV2-GFP-(FPC-CTD-5XMyc)
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