Drug Discovery & Development
A CRISPR on-off switch for genes controls expression without altering DNA
A new gene silencing tool allows scientists to switch genes on and off without altering genetic sequences. The tool, described in a paper published in Cell on April 9, uses a modified CRISPR-Cas9 system to introduce reversible epigenetic changes that control gene expression. Read More
BioTools Innovator seeks to boost startups developing life science tools
BioTools Innovator is calling for applications to its inaugural competition to advance innovation by accelerating the growth of startups developing life science tools. Kathryn Zavala, PhD, managing director, recently discussed the program with ScienceBoard.net. Read More
NIH details progress of SCGE consortium
The U.S. National Institutes of Health (NIH) Somatic Cell Genome Editing (SCGE) consortium has provided a detailed update, published in Nature on April 8, of projects to develop safer and more effective genome editing methods in somatic cells. Read More
Will preprints have a lasting effect on publishing beyond COVID-19?
During the COVID-19 pandemic, 25% of all COVID-19-related scientific manuscripts were shared on preprint servers, a steep increase from previous usage and relative to traditional peer-review journals. An international team of researchers explored the critical role of preprint servers in disseminating epidemic-related information in an article published in PLOS Biology on April 2. Read More
X-ray crystallography identifies drugs to be repurposed for COVID-19
A new large-scale study indicates x-ray crystallography can be used to find drugs that could be repurposed to target the SARS-CoV-2 main protease. In addition to identifying 37 potential drug candidates, the study, published in Science on April 2, revealed a new binding site on the SARS-CoV-2 main protease to which drugs can bind. Read More
2nd-gen stem cell-derived CAR gene therapy is more durable, effective against HIV
A novel second-generation chimeric antigen receptor (CAR)-based approach targeting HIV infection using the genetic modification of hematopoietic stem cells shows promise in preclinical models. The details of the study were published in PLOS Pathogens on April 1. Read More
New gene therapy could be effective in treating complex polygenic conditions
Scientists are applying gene therapy approaches in a new way by simultaneously administering a combination of cargos to treat complex polygenic neurodegenerative diseases with no single genetic cause. Details of the combination gene therapy in two animal models were detailed in a March 31 Science Advances article. Read More
Cancer anti-inflammatory drug has potential to treat severe COVID-19
A new study provides evidence that an inexpensive clinical-grade inhibitor that's already approved to treat cancer, called topotecan, can be used to treat severe COVID-19, even in the late stages of the disease. The findings were published in Cell on March 30. Read More
Prime editing creates desired gene mutations without collateral damage
A new gene editing tool called prime editing was demonstrated to efficiently create cell-specific knockout mice compared to traditional gene editing techniques. While both platforms successfully created mutations, prime editing did so without measurable on-target indels or off-targeting events, according to a study published in Genome Biology on March 16. Read More
Machine-learning system ranks most effective cancer drugs
Scientists successfully trained an ensemble of machine-learning (ML) algorithms to rank clinically relevant cancer drugs based on the drugs' predicted efficacy in reducing cancer cell growth. The study, reported in Nature Communications on March 25, suggests that ML may soon be widely used to predict the most appropriate treatment for individual patients with cancer. Read More
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