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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

Disease 142
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Second-Generation mRNA COVID-19 Vaccine Candidate, CV2CoV, Demonstrates Improved Immune Response and Protection in Preclinical Study

The Pharma Data

Better activation of innate and adaptive immune responses was achieved with CV2CoV, resulting in faster response onset, higher titers of antibodies, and stronger memory B and T cell activation as compared to the first-generation candidate, CVnCoV. “In Induction of innate immunity was investigated via specific cytokine markers.

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Decoding stem cells for personalised regenerative medicine

Drug Target Review

In research published in Scientific Reports , 1 investigators focused on mesenchymal stem cells (MSCs), known for their potential in treating cell defects and regulating immune responses. We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun.

RNA 128
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Study reveals how cancer immunotherapy may cause heart inflammation in some patients

Broad Institute

By Leah Eisenstadt November 6, 2024 Credit: Ricardo Job-Reese, Broad Communications Despite being one of the rarer complications from immune checkpoint inhibitors, myocarditis is the most deadly. Our results provide a more detailed picture of what’s happening in the heart and suggest intriguing new ways forward to improve patient care.”

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The role of CRISPR in microbiome engineering breakthroughs

Drug Target Review

When faced with a viral threat, bacterial cells developed an immune response by capturing and copying DNA fragments of viruses. It was also discovered that the Cas enzyme was responsible for DNA cleavage. Using a synthetic guide RNA (gRNA), scientists can target a specific DNA sequence and employ Cas9 to cut it.

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Revolutionary nanoparticles enable gene-editing in lungs

Drug Target Review

Scientists from the Massachusetts Institute of Technology (MIT) and the University of Massachusetts Medical School (UMass), US, have collaborated to create a novel type of nanoparticle that can deliver messenger RNA that encodes for beneficial proteins to the lungs.

RNA 98
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Unleashing the power of AI in target discovery and resilience against disease

Drug Target Review

In the quest to combat and eradicate diseases, scientists and researchers are increasingly turning to the immense potential of artificial intelligence (AI) as a tool. It was here that the Impact Medicine Fund was raised as a concept around wellness for those with disease, with the ambition of unlocking some of this vast potential.

Disease 75