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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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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. This allowed bacteria to recognise subsequent attacks and cleave the viral DNA to stop the viral infection. It was also discovered that the Cas enzyme was responsible for DNA cleavage.

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How the Classic TB Vaccine Treats Bladder Cancer – Zebrafish Avatars Reveal Mechanism

PLOS: DNA Science

One of the oldest and most successful immunotherapies is simpler: a tamed version of a classic vaccine, against the infectious disease tuberculosis (TB). Their report appears in Disease Models and Mechanisms. Eventually, a strain emerged that seemingly protected against a host developing the associated infectious lung disease.

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Designing a Better Probiotic. CRISPR Hubris?

PLOS: DNA Science

The molecular tools of CRISPR were borrowed and developed from the natural immune response of bacteria to viruses – bacteriophages – that infect them. CRISPR may make sense for replacing a single mutant gene, like the one behind sickle cell disease, for which a CRISPR-based gene therapy was recently approved.

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Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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Not all neoantigens are created equal

Drug Target Review

During the process of transformation from a normal cell into a cancer cell, a cell acquires a series of changes, or mutations, in its DNA. But DNA mutations can also result in changes to the proteins that are displayed on the surface of the cancer cell. Neoantigens are recognised as non-self and trigger an immune response.

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INOVIO Announces Publication of Phase 1 Data from its COVID-19 DNA Vaccine Candidate, INO-4800 in The Lancet’s EClinicalMedicine

The Pharma Data

” Dr. Stanley Plotkin , Professor Emeritus at The Wistar Institute, said, “INOVIO’s DNA vaccine appeared to be quite safe with few significant reactions but yet induced both antibody and T cell responses to SARS-CoV-2.” . PLYMOUTH MEETING, Pa. , mg and 2.0

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