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Genetic Engineering Publications - GEG Tech top picks
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BigField GEG Tech's insight:
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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Traumatic brain injury (TBI) is a major cause of mortality and morbidity, often causing lifelong disability for those who survive. Treatment is limited to supportive care, but stem cell therapy has received recent attention as a way to promote recovery for injuries to the central nervous system (CNS). In this study, researchers transplanted human neural stem cells (hNSCs) into the brains of mice modeled with TBI to investigate whether the hosts' immune systems and the stem cells acting in concert would enhance repair.
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The scientists report the results of subretinal transplantation of human embryonic-stem-cell (hESC)-derived retinal pigment epithelium in four Asian patients: two with dry age-related macular degeneration and two with Stargardt macular dystrophy. They were followed for 1 year. There was no evidence of adverse proliferation, tumorigenicity, ectopic tissue formation, or other serious safety issues related to the transplanted cells. Visual acuity improved 9–19 letters in three patients and remained stable (+1 letter) in one patient. The results confirmed that hESC-derived cells could serve as a potentially safe new source for regenerative medicine.
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In this work, the scientists assess the capacity of intra-arterial delivered autologous engineered canine CD133+ cells of restoring dystrophin expression in golden Retriever dystrophic dogs (GRMD) . This is the first demonstration of five-year follow up study, showing initial clinical amelioration followed by stabilization in mild and severe affected GRMD dogs. The occurrence of T-cell response in three GRMD dogs, consistent with a memory response boosted by the exon skipped-dystrophin protein, suggests an adaptive immune response against dystrophin.