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NY1972

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NY1972

Re: jondoeuk post# 132

Sunday, 05/22/2022 9:19:49 PM

Sunday, May 22, 2022 9:19:49 PM

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Donor T cells and donor genes to treat cancers?

Delivery of twin (AAV) capsids carrying 2 payloads: one with the nuclease to cut genomic DNA at the well-characterized PCSK9 locus and the other with a therapeutic donor gene, providing a potential path to permanent expression of a healthy gene

The long-term risks of off-target editing are unclear although
we are encouraged by the over 42 years of event free followup in 33 NHPs we have studied with this nuclease
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