A new study by scientists at UMass Medical School shows that using a technique called “nuclease-free” gene editing to correct cells with the mutation that causes a rare liver disease leads to repopulation of the diseased liver with healthy cells. Alpha-1 antitrypsin deficiency is an inherited disease that causes liver and lung damage; the Mueller...
Category: <span>Genetics</span>
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New genome-editing strategy could lead to therapeutics
Researchers at UMass Medical School have developed a genome-editing strategy to correct disease-causing DNA mutations in mouse models of human genetic diseases, according to research published in the Aug. 18 edition of Nature Biotechnology. Credit: CC0 Public Domain First author Dan Wang, Ph.D., instructor in microbiology & physiological systems, together with co-corresponding authors Guangping Gao, Ph.D., the Penelope Booth...
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New CRISPR technique skips over portions of genes that can cause disease
UNIVERSITY OF ILLINOIS AT URBANA-CHAMPAIGN CHAMPAIGN, Ill. — In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell’s internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building. This gives researchers a way not only to eliminate a mutated gene...
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Genes drive aging, making normal processes damaging
Ageing in worms mainly results from the direct action of genes and not from random wear and tear or loss of function, and the same is likely to be true in humans, according to research by UCL, Lancaster University and Queen Mary University of London scientists. IMAGE: THE DETERIORATIVE PART OF AGEING, CALLED ‘SENESCENCE’, IS THE...
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First grand project from Genome Project-write (GP-write) looks to develop ultra-safe virus-resistant cells
The leadership of the GP-write project – Jef Boeke from NYU Langone Medical Centre (NY), George Church from Harvard Medical School (MA), Andrew Hessel from Autodesk Inc (CA) and Nancy J Kelley from New York Genome Center (NY, all USA) – have announced their project’s latest developments. Updating the goals of their seminal GP-write paper, published...
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Parkinson’s gene affects more people than previously thought
New research finds that a gene previously thought to affect only a small percentage of Parkinson’s disease cases actually affects many more. The findings mean that treatments that are being developed for a small number of people may, in fact, benefit many more. Almost 1 million people in the United States and nearly 10 million people across the world...
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Gene therapy restores sense of smell in mice
Re-expressing a protein critical for the detection and perception of odors restores the function of the olfactory system in a genetic mouse model of lost hair-like cellular structures known as cilia, according to research published in JNeurosci. This may represent a promising therapeutic strategy for a group of human diseases that can cause loss of smell. Credit: Green...
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How diet modifies the correlation between genetics and obesity
A correlation between obesity and genetics has been found to be modified by diet, according to a scientific paper in the American Journal of Clinical Nutrition. A research group led by a U.S. Department of Agriculture (USDA) scientist found that a specific gene—APOA2—can result in a higher body mass index (BMI). The APOA2 gene encodes...
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MARRVEL: Integration of Human and Model Organism Genetic Resources to Facilitate Functional Annotation of the Human Genome
One major challenge encountered with interpreting human genetic variants is the limited understanding of the functional impact of genetic alterations on biological processes. Furthermore, there remains an unmet demand for an efficient survey of the wealth of information on human homologs in model organisms across numerous databases. To efficiently assess the large volume of publically...
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A simple guide to CRISPR, one of the biggest science stories of the decade
It could revolutionize everything from medicine to agriculture. Better read up now. One of the biggest and most important science stories of the past few years will probably also be one of the biggest science stories of the next few years. So this is as good a time as any to get acquainted with the powerful new...