Page 97 - PCC08

Germline genome editing
DNA editing of germline can be performed in
1. Stem cells
• Pluripotent stem cells differentiated into gametes to further give rise to an embryo (in vitro gametogenesis – IVG)
• Spermatogonial stem cells 2. Zygotes or Embryos
PCC Genetics - ESHRE BCN 2018
Gene correction Mouse model. Spermatogonial stem cells
• Engineered nucleases (ZFN) used to stimulate homologous recombination to correct a mutant gene in mouse germline stem cells (testicular derived cell cultures containing spermatogonial stem cells and progenitor cells).
• Gene-corrected cells maintained several properties of spermatogonial stem/progenitor cells including the ability to colonize following testicular transplantation.
• Proof of concept for genome editing in germline cells
Genome editing in mouse spermatogonial stem/progenitor cells using engineered nucleases. Fanslow, 2014
PCC Genetics - ESHRE BCN 2018
Page 91 of 102
PRECONGRESS COURSE 08 I BARCELONA, SPAIN – 1 JULY 2018 97