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A CRISPR on-off switch for genes controls expression without altering DNA
April 12, 2021 -- 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
April 9, 2021 -- 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
April 8, 2021 -- 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?
April 7, 2021 -- 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

Improved RNA sequencing method reveals new insights into small RNAs
April 6, 2021 -- Researchers have developed a new RNA sequencing method that can help discover many types of previously undetectable small RNAs. Details of the method, called panoramic RNA display by overcoming RNA modification aborted sequencing (PANDORA-seq), were published in Nature Cell Biology on April 5. Read More

X-ray crystallography identifies drugs to be repurposed for COVID-19
April 5, 2021 -- 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
April 2, 2021 -- 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
April 1, 2021 -- 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
March 31, 2021 -- 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
March 30, 2021 -- 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
March 29, 2021 -- 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

Breakthrough photochemistry method opens doors to new drugs
March 26, 2021 -- Medicinal chemists have succeeded in converting several classes of flat nitrogen-containing molecules into 3D structures that could form the basis for new drug candidates. The methods, published in Science on March 26, detail over 100 examples of the broad applicability of overcoming the conversion barrier through light-mediated energy transfer. Read More

Scientists connect gene to cancer metastasis for the first time
March 25, 2021 -- Researchers have identified new functionality of a previously underappreciated gene, leucine-rich repeat neuronal 4 C-terminal like (LRRN4CL), which is overexpressed in melanoma cells. They believe LRRN4CL plays an important role in cancer metastasis to the lungs and describe their findings in Communications Biology on March 23. Read More

Hydrogen maps of SARS-CoV-2 main protease point to effective repurposed drugs
March 24, 2021 -- Scientists have used atomic maps of hydrogen atoms to determine that the SARS-CoV-2 main protease acts in unexpected ways when it comes into contact with a drug inhibitor. The research, published in the Journal of Medicinal Chemistry on March 23, provides key insights for efforts to repurpose existing drugs to develop candidates for treating COVID-19. Read More

Scientists develop ultraspecific CAR T cells that kill only cancer cells
March 23, 2021 -- Scientists have devised a two-step engineering circuit that precisely targets solid tumors with chimeric antigen receptor (CAR) T cells. The engineering feat could be the next step in treating solid tumors using therapeutic T cells, a treatment that has remained out of reach for many cancer patients. The new method was detailed in a Science article on March 12. Read More

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