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In a human CEP290 IVS26 knock-in mouse model, EDIT-101 had strong productive editing rates within photoreceptor cells, effectively restored normal splicing of the CEP290 mRNA, and restored the production of the full-length CEP290 protein [63]

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With age, GSH levels decrease by up to 50% compared to young subjects, contributing to increased oxidative stress and chronic low-grade inflammation (inflammaging). In the anti-ageing field, GSH IV acts on three main levels: direct neutralisation of free radicals and protection of dermal collagen, modulation of inflammation through NF-B inhibition, and reduction of melanogenesis through tyrosinase inhibition with clinically observable effects on skin tone and luminosity. The most robust clinical evidence concerns neuroprotection (improvement of motor symptoms in Parkinson's disease) and protection from chemotherapy-induced neuropathy

A side effect that occurs in 0.1% of patients (1 in 1,000) has only a 28% chance of appearing in a 338-person trial