By SHARON BEGLEY | OCTOBER 21, 2019 A new form of the genome-editing tool CRISPR-Cas9 appears to significantly expand the range of diseases that could be treated with the technology, by enabling scientists to precisely change any of DNA’s four “letters” into any other and insert or delete any stretch of DNA — all more...
Category: <span>Genetics</span>
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Research improves understanding of mechanism of atrial fibrillation
by Baylor College of Medicine Atrial fibrillation is the most common heart arrhythmia in humans. This condition increases the risk of heart failure, stroke, dementia and death, and current treatments have suboptimal efficacy and carry side effects. Looking to identify clues that might lead to better treatments, a group headed by researchers at Baylor College...
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Super-precise new CRISPR tool could tackle a plethora of genetic diseases
The system allows researchers more control over DNA changes, potentially opening up conditions that have challenged gene-editors. Heidi Ledford For all the ease with which the wildly popular CRISPR–Cas9 gene-editing tool alters genomes, it’s still somewhat clunky and prone to errors and unintended effects. Now, a recently developed alternative offers greater control over genome edits — an advance...
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Scientists reveal novel oncogenic driver gene in human gastrointestinal stromal tumors
CHINESE ACADEMY OF SCIENCES HEADQUARTERS Sarcomas – cancers that arise from transformed mesenchymal cells (a type of connective tissue) – are quite deadly. Gastrointestinal stromal tumors (GISTs) are the most common human sarcoma and are initiated by activating mutations in the KIT receptor tyrosine kinase. Micro-GISTs are a smaller variation of clinical GISTs and are...
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New universe of miniproteins is upending cell biology and genetics
By Mitch LeslieOct. 17, 2019 , 2:00 PM Mice put human runners to shame. Despite taking puny strides, the rodents can log 10 kilometers or more per night on an exercise wheel. But the mice that muscle biologist Eric Olson of the University of Texas Southwestern Medical Center in Dallas and colleagues unveiled in 2015...
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Russian ‘CRISPR-baby’ scientist has started editing genes in human eggs with goal of altering deaf gene
Denis Rebrikov also told Nature that he does not plan to implant gene-edited embryos until he gets regulatory approval. David Cyranoski An earlier version of this story and its headline incorrectly stated that Denis Rebrikov had started editing eggs from a woman with a mutation that causes deafness. Russian biologist Denis Rebrikov has started gene editing in eggs donated by women who can...
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Gene therapy for Duchenne muscular dystrophy safely preserves muscle function
by Perelman School of Medicine at the University of Pennsylvania A gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease in both small and large animal models, according to a first-of-its-kind study from Penn Medicine researchers. The...
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NIH researchers create new viral vector for improved gene therapy in sickle cell disease
‘Forward-oriented’ design might boost treatment effectiveness and broaden use NIH/NATIONAL HEART, LUNG AND BLOOD INSTITUTE Researchers at the National Institutes of Health (NIH) have developed a new and improved viral vector–a virus-based vehicle that delivers therapeutic genes–for use in gene therapy for sickle cell disease. In advanced lab tests using animal models, the new vector was up to 10 times more efficient at incorporating corrective genes into bone marrow stem...
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A comprehensive atlas of genetic regulation of lipid metabolism published
UNIVERSITY OF HELSINKI An international research team has identified several novel genetic variants associated with plasma levels of lipid species and cardiovascular disease risk in humans. The study demonstrates that genetic studies focusing on circulating molecular lipid levels over traditional lipid measures can help improve cardiovascular risk prediction and treatment. The results of the study...
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New mechanisms that regulate pluripotency in embryonic stem cells are discovered
by Karina Toledo, FAPESP Embryonic stem cells (ESCs) can give rise to many types of tissues and organs. At the turn of the century, these cells were believed to offer hope of treatment for several health problems, but as research advanced, scientists realized that understanding and controlling the behavior of ESCs would be a more...