dna

A Better Carrier – New Gene-Delivery Therapy Restores Partial Hearing, Balance in Deaf Mice

Using a novel form of gene therapy, scientists from Harvard Medical School and Massachusetts General Hospital have managed to restore partial hearing and balance in mice born with a genetic condition that affects both. The new model overcomes a long-standing barrier to accessing hair cells, the delicate sensors in the inner ear that capture sound and head movement and convert them…

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Penn Scientists Use CRISPR for First Time to Correct Clotting in Newborn and Adult Mice

CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations. For the first time, researchers from the Perelman School of Medicine at the University of Pennsylvania have developed a dual gene therapy approach to deliver key components of a CRISPR/Cas9-mediated gene targeting system to mice to treat hemophilia B. This disorder is also called factor…

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Scripps Florida Scientists Uncover New Facets of Zika-Related Birth Defects to Help Develop Treatment

In a study that could one day help eliminate the tragic birth defects caused by Zika virus, scientists from the Florida campus of The Scripps Research Institute (TSRI) have elucidated how the virus attacks the brains of newborns, information that could accelerate the development of treatments. The study, led by TSRI Associate Professors Hyeryun Choe and Damon Page, was published…

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DNA mapping tool helps scientists better understand how genes are regulated

Scientists have devised a powerful new tool for understanding how DNA controls gene activity in cells. The tool allows researchers to map at high resolution, across large swaths of a cell’s genome, which DNA nucleotides work to regulate gene activity. “This is the first method that enables us to simultaneously test thousands of human DNA regulatory regions for their ability…

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