OXB-204 / Oxford Biomedica - LARVOL DELTA

[VIRTUAL] OXB-204: A lentiviral vector-based gene therapy to correct all mutations in CEP290. (ESGCT 2021) - No abstract available

Gene Therapies • CEP290

OXB-204: A Lentiviral Vector-based Gene Therapy Approach to Treat Leber Congenital Amaurosis 10 Due to Mutations in CEP290 (ARVO 2020) - "Building on our clinical data from OXB-201, (Campochiaro et al, 2017) which demonstrated long term transgene expression out to 6 years following a single subretinal administration to patients, CEP290 was cloned into the same clinical vector backbone driven by a constitutive CMV promoter.Results Lentiviral vectors were generated and shown to express the full length CEP290 protein. To demonstrate CEP290-dependent cilia defects, endogenous expression of CEP290 in ARPE-19 was knocked down using CEP290-specific shRNA. Rescue of the cilia defects obtained in the ARPE-19 CEP290-knock down cells with OXB-204 was achieved.Conclusions OXB-204, a lentiviral vector encoding a full-length copy of human CEP290 was capable of rescuing CEP290-knock down cilia defects in vitro."