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Gene editing DNA deletion sizes reduced with new fusion method developed at WFIRM

Scienmag

WINSTON-SALEM, NC – May 2, 2022 — Wake Forest Institute for Regenerative Medicine (WFIRM) scientists working on CRISPR/Cas9-mediated gene editing technology have developed a method to increase efficiency of editing while minimizing DNA deletion sizes, a key step toward developing gene editing therapies to treat genetic diseases.

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Why early participant engagement is now a top priority in genetic disease research

pharmaphorum

In 2016, scientists behind a study called the Resilience Project analysed genetic data from 589,000+ people and found 13 adults who carried genetic variants that should have resulted in serious – even deadly – childhood disease, but who were apparently healthy.

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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…

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Ancient viruses may hold key to gene therapy treatments

Drug Discovery World

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . It was led by scientists at the MRC-University of Glasgow Centre for Virus Research (CVR) and University of Massachusetts Chan Medical School. .

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Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

CAMP4 is exploiting these regulatory interactions to specifically control the expression of genes tied to disease. So, regRNAs haven’t been exploited previously because scientists have only recently understood their role in regulating gene expression. What diseases are you currently targeting? Can you give some examples?

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Why genomic healthcare data matters in the development of new therapies 

Drug Discovery World

From a general healthcare perspective, access to this sort of data is invaluable, but this is particularly noteworthy in that DNA tests have historically been limited in these regions on account of cost and availability of practitioners, services and resources.

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Personalising whole genome sequencing doubles diagnosis of rare diseases 

Drug Discovery World

In 2018, the UK’s department of health announced an NHS Genomic Medicine Service, which allows patients with rare diseases to have their entire genetic code read in the hope of providing a much-needed diagnosis. That said, some people with rare genetic diseases remain without a molecular diagnosis after their genome is analysed. . “We

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