Alex Monteith

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gene editing in vivo gene insertion mutation-agnostic curative therapies liver disorders rare diseases orphan drug development urea cycle disorders otc deficiency citrullinemia type 1 phenylketonuria genetic medicine clinical trials aav vectors arcus nuclease genome editing ivd technology pediatric drug development biopharma fda approvals genetic therapies therapeutic outcomes gene therapy safety & efficacy investigational new drug genomic medicine healthy gene insertion disease-causing mutations long-term expression gene therapy manufacturing clinical response pharmacokinetics neonatal onset conditions patient management gene replacement genetic mutations biological markers healthcare innovation immunosuppressive therapy metabolic disorders toxic ammonia buildup diagnostic tools treatment protocols biologic markers therapeutic interventions health care health wellness & fitness hospital & health care