In demonstrations in cell lines, the technique has a similar efficacy to CRISPR-Cas9, but fewer off-target effects.
2020-06-02Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.
2020-06-01In less than a decade since its adaptation to a genome-editing technique, CRISPR-Cas9 has been used on lab animals and cells around the world, as well on human cells that are already being tested in clinical trials to treat disease. Limitations such as off-target edits are widely …
2020-05-29When the human papillomavirus enters a cervix, it doesn’t lyse cells or cause inflammation. While some strains can cause genital warts, in most cases the body clears the virus without much fuss. But “in an unfortunate number of people, the virus gets stuck,” says Nigel McMil…
2020-05-28CRISPR genome editing tools are driving innovations in precision genetic medicine. However, in vivo delivery of CRISPR components for treating diseases remains a challenge as researchers strive to eliminate or reduce off-target effects, provide transient editing systems, and incre…
2020-05-27Researchers slowed disease progression in the mice by injecting two different viral vectors, each containing one part of the DNA encoding the Cas9 protein, to edit the causative gene.
2020-05-25Horizon Discovery describes how short hairpin RNA is advancing the development of chimeric antigen receptor T-cell therapies
2020-05-22Mammoth Biosciences is responding to the urgent need for improved and more accessible testing for COVID-19 by rapidly reconfiguring the company’s CRISPR-based diagnostics platform to detect the presence of SARS-CoV-2. In April, the company published a study demonstrating the pow…
2020-05-21MilliporeSigma said it has been granted two U.S. patents toward CRISPR-Cas9-assisted genome-editing, the company's 25th and 26th CRISPR patents worldwide and third and fourth in the U.S. [Janet Iwasa for the Innovative Genomics Institute at UC Berkeley]
2020-05-20