Drug Discovery & Development
Potential of gene editing for clinical use highlighted at ASGCT 2021
From base editing to in vivo CRISPR therapeutics and CRISPR-modified bacteriophages, scientists discussed innovations in preclinical research that have allowed them to advance these unique products to the clinic during a scientific symposium at the 2021 American Society of Gene & Cell Therapy (ASGCT) virtual meeting. Read More
Nobel Prize winner Doudna shares perspectives on future of CRISPR gene editing
Small science can translate into big discoveries, according to Nobel Prize laureate Jennifer Doudna, PhD. She discussed how her curiosity in understanding CRISPR led to the gene editing revolution in a talk at the 2021 American Society of Gene & Cell Therapy virtual meeting. Read More
Variant-resistant COVID-19 vaccines could be effective, cheap to make
Researchers have collaborated to create a new COVID-19 vaccine platform containing antigens against the fusion protein of the SARS-CoV-2 virus that could be effective against variants of the virus. The research was published online on April 15 in the Proceedings of the National Academy of Sciences. Read More
AACR 2021: How machine learning and artificial intelligence are transforming cancer research
The use of digital tools has been incorporated into many areas of cancer research. However, experts agree that machine learning and artificial intelligence are poised to transform drug discovery efforts even further in the near future. Several scientists discussed case studies of how machine learning can be used in cancer biology and drug discovery efforts during the virtual American Association for Cancer Research (AACR) 2021 meeting. 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
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
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