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CRISPR in the Clinic

addgene Blog

In 2014, we wrote a blog post on CRISPR’s potential for correcting monogenetic diseases. There can be no doubt that CRISPR/Cas9 technology has been a breakthrough for the genome-editing field and the greater scientific community.

Therapies 128
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When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

FDA Law Blog: Drug Discovery

For instance, the December 2024 guidance goes beyond the 2014 Expedited Programs for Serious Conditions Drugs and Biologics guidance (the last to deal substantively with accelerated approval) to describe FDAs expectation that sponsors take a proactive approach to ensuring confirmatory trials are completed within specified timelines.

FDA 105
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A new viral surveillance system in West Africa is showing the world how to prevent the next pandemic

Broad Institute

By Allessandra DiCorato April 25, 2024 As night fell on July 20th, 2014, genomicist Christian Happi was at home with his family in Lagos, Nigeria when he received a phone call he would never forget. He started to imagine how the deadly and contagious disease, if confirmed, might spread to half the city’s population. Happi felt chilled.

Virus 132
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Viral Vectors 101: What is a Virus (and a Viral Vector)?

addgene Blog

Viruses are simple – a genome packaged in a protein shell (Taylor, 2014). Yet these simple, small particles have quite the outsized impact – and not just on the disease front. They’re so simple that we can’t even decide if they’re alive or not.

Virus 97
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The science of ageing and restoring healthspan

Drug Target Review

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

Science 145
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Innovative research on schistosomiasis-associated colorectal cancer (SA-CRC) yields unique insights into genetic mutations and treatment implications

SCIENMAG: Medicine & Health

In a study published in the journal Genes & Diseases, researchers from Naval Medical University and Soochow University conducted an in-depth investigation into the genomic landscape of schistosomiasis-associated colorectal cancer (SA-CRC).

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Largest-ever genetic study of epilepsy finds possible therapeutic targets

Broad Institute

Epilepsy also has several subtypes, and while one group called developmental encephalopathies have been connected to several genes, other forms of the disease are less well understood. The exomes revealed connections between disease risk and several genes involved in the transmission of signals across the synapses between neurons.

DNA 133