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Customized gene therapies successfully target rare eye diseases
September 8, 2020 -- Can gene therapies prove effective for the treatment of rare genetic diseases? One company may have found a solution that incorporates a high degree of customization, manufacturing expertise, and years' worth of knowledge. Discuss

Experts draw lessons from FDA's hydroxychloroquine EUA controversy
September 7, 2020 -- Public health and regulatory experts advocated for changes to the U.S. Food and Drug Administration (FDA) emergency use authorization (EUA) process in a viewpoint published online August 31 in the Journal of the American Medical Association. They offer a set of recommendations to improve the process going forward as more COVID-19 vaccines and treatments emerge. Discuss

New CRISPR-repressor system improves efficiency of gene therapies
September 4, 2020 -- Researchers have developed a CRISPR-based system that can simultaneously provide transcriptional control and gene editing on demand to improve the efficacy of gene therapies. The details were published in Nature Cell Biology on September 3. Discuss

New gene therapy destroys latent oral herpes in mice
September 4, 2020 -- Researchers recently reported that they were able to eliminate latent herpes simplex virus 1 in mice using a new gene editing technique that targets the root cause of oral herpes. The findings were published on August 18 in Nature Communications. Discuss

Protein engineering creates highly potent experimental SARS-CoV-2 vaccine
September 3, 2020 -- A bioengineering technique that utilizes various naturally occurring sequences and de novo design of messenger RNA (mRNA) components was applied to develop an mRNA-based vaccine that could be highly effective against SARS-CoV-2, according to a new article published in Advanced Materials on September 2. Discuss

Detecting SARS-CoV-2 in blood may be early indicator of severe disease
September 2, 2020 -- A blood test that measures SARS-CoV-2 RNA when patients are admitted to the hospital with COVID-19 symptoms can be a powerful diagnostic tool to predict how severe their disease will be, according to a study published in Clinical Infectious Diseases on August 28. Patients without viral RNA in their blood have a good chance at rapid recovery, concluded researchers from Karolinska Institutet and Danderyd Hospital. Discuss

New small-molecule drug targets hard-to-reach cancer-linked enzyme
September 1, 2020 -- Researchers have developed a new class of small-molecule drugs that show promise against a subset of pediatric leukemia with the NUP98-NSD1 chromosomal translocation. The findings were published in Nature Chemical Biology on August 31. Discuss

Gateway receptor for SARS-CoV-2 helps explain variability of COVID-19
August 31, 2020 -- A wide variety of symptoms and organs are involved in SARS-CoV-2 infection. This phenomenon could be explained by the distribution of the virus's gateway receptor, which is found in tissues throughout the body. Characterization of angiotensin-converting enzyme 2 expression is detailed in a recent Cellular and Molecular Neurobiology article. Discuss

Study investigates pharmacogenomics of COVID-19 therapies
August 31, 2020 -- Human genetic variation may alter the interactions of drugs being used to treat patients with COVID-19 symptoms, resulting in a range of clinical responses -- from no effect to high toxicity in some patients, according to a study published in Nature Genomic Medicine on August 18. Discuss

New mouse-adapted model helps accelerate COVID-19 vaccines, therapies
August 28, 2020 -- A new mouse-adapted SARS-CoV-2 model developed with engineered virus can help accelerate vaccine and therapeutic candidates to clinical trials. The results of the preclinical studies were published in Nature on August 27. Discuss

NIH establishes Centers for Research in Emerging Infectious Diseases
August 27, 2020 -- The National Institute of Allergy and Infectious Diseases, part of the U.S. National Institutes of Health (NIH), has awarded 11 grants for a first-year total value of approximately $17 million to establish the Centers for Research in Emerging Infectious Diseases. Discuss

Molecular barcoding of DNA identifies rare mutations in stem cells
August 27, 2020 -- Scientists have developed a new next-generation sequencing technique using molecular barcodes that can accurately detect a single genetic mutation in a pool of 10,000 cells. The researchers applied the approach to CRISPR-Cas9 genome editing to search for undesirable mutations caused by the platform. The research and methods were published in Genome Biology on August 24. Discuss

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