High-Fidelity CRISPR-Cas9 Nucleases Virtually Free of Off-Target Noise
If a Cas9 nuclease variant could be engineered that was less grabby, it might loosen its grip on DNA sequences throughout the genome except those
If a Cas9 nuclease variant could be engineered that was less grabby, it might loosen its grip on DNA sequences throughout the genome except those
Highlights Small-molecule inhibition of RAD52 activity in vitro and in vivo Small-molecule dimerization of RAD52 undecamer rings Small-molecule disruption of RAD52 ring superstructures Small-molecule inhibition of RAD52
Chinese stem-cell scientists have welcomed long-awaited measures that, state media claim, will rein in rogue use of stem cells in clinics while allowing research. The
Scientists have used two methods to generate patient-specific pluripotent stem cells with normal mitochondria for people with defects in these organelles, according to a study
Researchers at the Salk Institute report the discovery of a new type of pluripotent stem cell whose identity is tied to its location in a
Scientists in Korea have injected human embryonic stem cell (hESC)-derived retinal support cells into the eyes of four men with macular degeneration, according to a
Patients with Leber’s congenital amaurosis (LCA), a genetic blindness disorder caused by a mutation in the retinal pigment epithelium 65 (RPE65) gene that encodes a
In a world first, Chinese scientists have reported editing the genomes of human embryos. The results are published1 in the online journal Protein & Cell
Mothers may one day be able to prevent their children from inheriting mitochondrial defects. Therapies that modify diseased eggs are inching closer to the clinic,
In the wake of the first ever report that scientists have edited the genomes of human embryos, experts cannot agree on whether the work was
A two-armed molecule has been developed that effectively locks HIV in a kind of miniature full nelson, preventing the virus from wrestling its way into
A common genetic blood disorder has been corrected in cultured stem cells by using a cutting-edge genome-editing technique. The disorder β-thalassaemia is characterized by reduced
Gene therapy could soon join the electronic pacemaker as a treatment for a weak heart. By inserting a specific gene into heart-muscle cells, researchers can
Monoallelic point mutations of isocitrate dehydrogenase type 1 (IDH1) are an early and defining event in the development of a subgroup of gliomas1, 2, 3 and other types
A few patients with a rare degenerative eye disease, called choroideremia, had partially restored vision following a gene therapy trial to deliver a healthy copy
Most children with spinal muscular atrophy (SMA) will never jump rope, play tag, or even walk because a genetic deletion will provoke the gradual destruction
Genome Research, DOI: 10.1101/gr.122044.111. Denis A. Smirnov, Lauren Brady, Krzysztof Halasa, Michael Morley, Sonia Solomon3 and Vivian G. Cheung. “Genetic variation in radiation-induced cell death“.
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