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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 189
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Unexplored genomic control regions yield the key to finding causes of rare disease

Medical Xpress

Scientists have discovered the cause of a rare condition within a part of the genome that has been largely unexplored in medical genetics.

Genome 98
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STAT+: Ahead of genome summit in London, questions linger about CRISPR baby scandal

STAT News

Next week, hundreds of scientists from around the world will convene in London for an international summit on genome editing. That technology, which enables scientists to easily excise, alter, or replace specific sections of DNA, was awarded the 2020 Nobel Prize for Chemistry. Continue to STAT+ to read the full story…

Genome 111
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Scientists use genomics to counter antimicrobial resistant typhoid

Drug Discovery World

Genome sequencing has been used to study typhoid fever in a study in Zimbabwe and understand how the disease has evolved to be resistant to treatment. Working with local health authorities in Harare, they sequenced the genomes of 85 S. Typhoid fever claims over 135,000 lives annually. Typhi population beforehand.

Genome 52
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Genetically Modifying Bats Could Prevent the Next Pandemic, Scientists Say

BioSpace

The next COVID pandemic could be prevented by using a gene drive to preemptively edit the genome of bats to prevent them from becoming hosts for coronaviruses, according to a proposal by scientists from Israel’s Interdisciplinary Center Herzelia and the National Institutes of Health.

Scientist 103
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STAT+: Moderna expands CRISPR gene editing research with ElevateBio partnership

STAT News

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.

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Uncovering packaging impurities with rAAV genome sequencing

Drug Discovery World

Join DDW for this exclusive free event, ‘ rAAV genome sequencing with Oxford Nanopore uncovers transgene integrity and packaging impurities ’. Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The event will be supported by Oxford Nanopore Technologies.