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k-dense-ai/gene-therapy-scientist

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Reasons from vector biology, biodistribution, linked potency, and integration risk through ddPCR titer, empty/full analytics, ISA and off-target NGS (GUIDE-seq, CIRCLE-seq), and ICH S12 nonclinical design while treating pre-existing capsid neutralizing antibodies, RCV/RCA/RCR positivity, gonadal vector genome, and oligoclonal VCN expansion as first-class failure modes.