Doctors in Oregon delivered the gene editing machinery behind the retina in hopes of treating an inherited form of blindness, according to the companies that developed the therapy.
2020-03-16Barth syndrome is a rare metabolic disease in boys caused by mutation of a gene called tafazzin or TAZ. It can cause life-threatening heart failure and also weakens the skeletal muscles, undercuts the immune response, and impairs overall growth. There is no cure or specific treatm…
2020-03-13Using a novel single-cell RNA sequencing approach, a team from the Oncode Institute has uncovered new details about the rates of messenger RNA synthesis and degradation in mammalian cells.
2020-03-11The potential that gene-editing tools such as CRISPR-Cas9 hold for eliminating disease and genetic disorders is immense.
2020-03-10CRISPR-based genome editing relies on creating a double-strand DNA break to form an indel that can either cause frameshift mutations and disrupt the gene or enable the uptake of a donor template sequence.
2020-03-09Rough-and-ready Cas9 enzymes are powerful and popular CRISPR tools, but they’re hardly the be-all, end-all of genome editing
2020-03-06CRISPR therapeutics for sickle-cell disease, says IDT, need fewer off-target effects and more on-target efficiency
2020-03-05Advanced cancer patients treated with immune cells that underwent CRISPR-mediated gene editing exhibited no serious side effects, New Scientist reports. It notes that concerns about the treatment particularly revolved around whether unintended CRISPR cuts occur and turn those cell…
2020-03-04In search of new ways to sequence human genomes and read critical alterations in DNA, researchers say they have successfully used the gene cutting tool CRISPR to make cuts in DNA around lengthy tumor genes, which can be used to collect sequence information.
2020-03-03