Peter Ghoroghchian

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biotechnology research gene therapy precision delivery aav technology cns treatment therapeutic genes novel capsid variants potency immunogenicity toxicology high throughput screening disease-specific targeting one-time gene therapy tissue-specific administration huntingtons disease parkinsons disease genetic hearing loss pediatric applications clinical techniques cell-specific targeting treatment paradigms patient quality of care side effects reduction improved efficacy toxic payload delivery lysomal storage disorders neurodegenerative diseases cross-correction therapy brain microvascular targeting healthcare innovation administration routes caregiver support biotechnology advancements clinical research high-potency treatments patient outcomes neutralized immune responses therapeutic candidates disease modification healthcare equity clinical efficacy genetic interventions translational medicine leading scientists medical professionals research publications partner collaborations development pipeline rehabilitation therapies innovative medicines