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Leveraging Genetic Testing for Enrolling Rare Disease Trials

Worldwide Clinical Trials

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

Genetics 168
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We’ve Discovered A Subtle Genetic Imbalance That May Drive Aging

AuroBlog - Aurous Healthcare Clinical Trials blog

Scientists have found an extremely subtle twist in the genetics of aging cells, one that seems to make them increasingly less functional as time goes on.

Genetics 172
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Scientists discover key genes behind insect migrations

Scienmag

Scientists have identified more than 1,500 genetic differences between migratory and non-migratory hoverflies. Credit: Will Hawkes Scientists have identified more than 1,500 genetic differences between migratory and non-migratory hoverflies.

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Scientists develop genetic ‘monitors’ that detect when genes are active

Scienmag

New genetic sensors, developed by scientists at University of Warwick and Keele University, could function as a lab test device and even as a live monitoring system inside living cells The innovative system can detect when a specific gene in a cell is active – instead of only detecting its presence The technology is based […].

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STAT+: On the long road to treating Huntington’s genetic stutter, scientists return to overlooked clues

STAT News

The scientists sent the blood 950 miles east to Massachusetts General Hospital in Boston, to a tiny lab (recently converted from a storeroom) where a 28-year-old postdoc named James Gusella and his 23-year-old research technician, Rudolph Tanzi, got to work.

Genetics 111
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Scientists identify new gene involved in autism spectrum disorder

Scienmag

Forward genetics pinpoints gene linked to ASD involving severe speech impairment and opens door to search for more mutations, future treatments Credit: Photo taken by Brian Coats for UT Southwestern Medical Center DALLAS – Dec.

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Cell and gene therapies: why advanced medicines call for specialised logistics

Pharmaceutical Technology

Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine. A new frontier in cancer research.