Drug Discovery & Development
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
Breakthrough photochemistry method opens doors to new drugs
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
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
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
Gene therapy based on transcription factors could be effective against Alzheimer's
A type of gene therapy using transcription factors to target DNA has been shown to dramatically reduce levels of the harmful protein tau in preclinical mouse models of Alzheimer's disease. The findings, published in Science Advances on March 19, suggest that the therapy could easily translate to the clinic and be effective in humans. Read More
What pregnant women need to know about COVID vaccines
Addressing questions about COVID-19 vaccines for pregnant and postpartum women, a leading maternal-fetal medicine specialist explained that it's likely best to get vaccinated as soon as they are eligible. Read More
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