Module 9 2021

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Gene editing: creation of biallelic site specific DSB ● Meganuclease, ZFN, TALENS and CRISPR/Cas9 ● NHEJ to knock out specific genes via Insertion / Deletion (indels) ● HDR for gene correction via point mutation, safe harbor gene addition and targeted gene addition. CRISPR/Cas9: easy adaptable and scalable site specific RNA-DNA base hybridization – SCD correction via BCLA-11 knock out, HIV correction via CCR5 Knock out – Targeted gene correction for DMD – Germline engineering

Novartis Pharma AG- Business use only - not for further distribution

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