Genome editing technologies, such as CRISPR-Cas9, enable the generation of isogenic hiPSC lines with which to examine the functional contribution of single variants within any genetic background. Studies of these rare variants using hiPSCs have the potential to identify commonly disrupted pathways in schizophrenia and allow for the identification of new therapeutic targets.
While porcine organs are considered promising, their use has been checked by concerns about transmission of porcine endogenous retroviruses (PERVs) to humans. Here, the scientists describe the eradication of all PERVs in a porcine kidney epithelial cell line (PK15). Using CRISPR-Cas9, they disrupted all 62 copies of the PERV pol gene and demonstrated a >1000-fold reduction in PERV transmission to human cells using their engineered cells. This study shows that CRISPR-Cas9 demonstrates the possibility that PERVs can be inactivated for clinical application to porcine-to-human xenotransplantation.
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Genome editing technologies, such as CRISPR-Cas9, enable the generation of isogenic hiPSC lines with which to examine the functional contribution of single variants within any genetic background. Studies of these rare variants using hiPSCs have the potential to identify commonly disrupted pathways in schizophrenia and allow for the identification of new therapeutic targets.
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