Remove Containment Remove Genetic Disease Remove Genome Remove Research
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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. Giving participants something in return. with their priorities.

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New research begins to write the rules on CRISPR activation

Drug Discovery World

UK researchers, from the Wellcome Sanger Institute and collaborators, have used human stem cells and neurons to investigate what features influence how well CRISPR activation works for different sets of genes. The post New research begins to write the rules on CRISPR activation appeared first on Drug Discovery World (DDW).

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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. . The research focuses on a group of small, ubiquitous viruses called ‘parvoviruses’ (from the Latin word ‘ parvus’ meaning ‘small’, ‘puny’ or ‘unimportant’).

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. The research was published earlier this month in the journal Cell.

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Searching for answers in rare epilepsy

pharmaphorum

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 83
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Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. The research was published earlier this month in the journal Cell. pyogenes dCas9.

DNA 52
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Searching for answers in rare epilepsy

pharmaphorum

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 66