Remove Immune Response Remove RNA Remove Therapies
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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. In the lab, they labelled RNA molecules with fluorescent markers, enabling them to easily locate them within individual cells.

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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. “As

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Cell and gene therapy development moves into cardiac indications

Drug Target Review

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

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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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Developing an mRNA therapy for Duchenne muscular dystrophy

Drug Target Review

There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. AAV-based therapies often can’t be dosed more than once due to concerns about an immune response. Of note, mRNA is redosable as opposed to AAV.

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A research team searches for every gene that helps tumors evade immunotherapy

Broad Institute

His mother had a presentation of the disease that suggested her immune system was already on the job. But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way.

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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. The study appears in Nature Biotechnology.

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