Germline gene therapy introduces intentional genomic alteration into zygotes, totipotent embryonic stem cells, or gamete precursors contributing to both somatic tissues and gonadal germline, establishing heritable change transmitted through fertilization. Using CRISPR Cas9 ribonucleoprotein comprising guide RNA complementary to 20 bp target adjacent to PAM and Cas9 nuclease with HNH and RuvC domains, double-strand break created in totipotent cell repaired by homology-directed repair incorporating donor template correcting mutation throughout embryo. Every tissue including testes and ovaries ca
Ref:
Nature Rev Genet Germline Editing Ethics 2017; NIH StatPearls Germline Therapy; UNESCO Bioethics Declaration https://www.nature.com/articles/nrg.2017.52.