Module 9 2021

Applications ● Beta-thalassemia and sickle cell dieseaes (BCLA11) ● HIV infection (CCR-5) ● off the shelf allogeneic cells (HLA and B2-macroglobulin): UCAR-T Ethical concerns ● Genome editing of human germline cells ● Transmission to the progeny introducing modified genomes in the human pool ● Uncontrolled use of CRISPR/Cas9 ● CCR-5 Knock out in two embryos to prevent HIV transmission by He Jankui from Shanghai University ● A 5-year moratorium proposed in an open letter to Nature on March ● EBFPIA/ EBE report in preparation Genome editing: potential applications and novel issues

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

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