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For children with spinal muscular atrophy (SMA), gene therapy with onasemnogene abeparvovec (Zolgensma, Novartis) provides long-lasting benefits with a favorable safety profile, new long-term follow-up data show. At a median of 5.2 years since receiving the approved therapeutic dose, onasemnogene abeparvovec provided “sustained, durable efficacy, with all patients alive and without the need for permanent […]
ZURICH (Reuters) – Vaccines from AstraZeneca, Russia’s Gamaleya Institute and Johnson & Johnson fight the coronavirus with another virus, leaving scientists concerned the shots may lose potency if annual inoculations become necessary to fight new variants. So-called viral vector shots – also used by several Chinese COVID-19 vaccine developers – use harmless modified viruses as […]
A new study using mass spectrometry is helping piece together what happens when DNA that has been sensitized by the oncology drug 5-fluorouracil is subjected to the ionising radiation used in radiotherapy. The anti-cancer drug 5-fluorouracil (5FU) acts as a radiosensitizer: it is rapidly taken up into the DNA of cancer cells, making the cells […]
Neurodevelopmental disorders arising from rare genetic mutations can cause atypical cognitive function, intellectual disability, and developmental delays, yet it is unclear why and how this happens. Scientists suspected a mutation in a complex of proteins could be the culprit for a group of rare genetic disorders and, now, Salk Institute researchers have identified the molecular […]
A new discovery by researchers at Columbia University Vagelos College of Physicians and Surgeons could fix one of the major shortcomings of current gene-editing tools, including CRISPR, and offer a powerful new approach for genetic engineering and gene therapy. Their new technology, called INTEGRATE, harnesses bacterial jumping genes to reliably insert any DNA sequence into […]
A large, multi-ethnic genome-wide association study (GWAS) of asthma identified novel associations with potential relevance for asthma susceptibility in older adults of diverse racial backgrounds. The study, “Large-scale, multi-ethnic genome wide association study identifies novel loci contributing to asthma susceptibility in adults,” appears in the April issue of the Journal of Allergy and Clinical Immunology. […]
DNA is small. Really, really, small. So, when researchers want to study the structure of a single-stranded DNA, they can’t just pull out their microscopes: they have to get creative. In a study published this week in Scientific Reports, researchers from Japan’s Osaka University explain how they came up with a really small solution to […]
A nationwide consortium of researchers has identified the first genetic defect linked to biliary atresia, a mysterious liver disease that is the leading cause for liver transplantation in children. The results were published in the journal Hepatology. The causes of biliary atresia were unknown, so this is a major advance that will move the field […]
Cholesterol carried in high-density lipoprotein particles, or HDL cholesterol, has been dubbed the good cholesterol, because people whose HDL levels are high have a lower risk of developing heart disease. That link was first established in 1977 and has been confirmed over and over in epidemiological studies. But in the past 15 years, a string […]
Inside the cell, DNA is tightly wrapped around proteins and packed in a complex, 3D structure that we call “chromatin.” Chromatin not only protects our genetic material from damage, but also organizes the entire genome by regulating the expression of genes in three dimensions, unwinding them to be presented to the cell’s gene-expression machinery and […]