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#germline gene therapy

2 public questions tagged with this topic.

Gene Therapy

Germline gene therapy is controversial because:

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.

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Gene Therapy

Somatic gene therapy differs from germline gene therapy because somatic therapy:

Somatic gene therapy restricts genetic modification to differentiated somatic lineages such as hematopoietic stem cells, hepatocytes, myofibers, retinal pigment epithelium, or T lymphocytes, preserving integrity of primordial germ cells in gonadal ridges. Recombinant lentiviral vectors pseudotyped with VSV-G bind LDL receptor, entering CD34+ stem cells after cytokine stimulation with SCF, TPO, FLT3L that drives cell cycling required for reverse transcription and integration. Integrated provirus flanked by LTRs replicates synchronously with host chromosomes during mitosis, ensuring inheritance

Ref: CCMO Netherlands Somatic vs Germline Therapy; MedlinePlus Somatic Gene Editing; Watson Mol Biol Gene Chap 12.

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