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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Its a huge privilege to have the opportunity to point these powerful new tools at our disease.

Disease 144
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Gene therapy gets a turbo boost

Science Daily: Pharmacology News

For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. But a major hurdle has been finding a safe and efficient way to deliver those genes.

Therapies 307
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New gene delivery vehicle shows promise for human brain gene therapy

Science Daily: Pharmacology News

In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.

Therapies 320
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New gene therapy for muscular dystrophy offers hope

Science Daily: Pharmacology News

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

Therapies 307
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Early Research Points to Possible New Parkinson's Drug

Drugs.com

19, 2024 -- Parkinson's is a relentless disease for which few treatments, and no real cure, exists. Now, researchers say they are on the trail of a potential new therapy for the disabling neurological illness.It's early research. THURSDAY, Dec.

Research 264
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A new switch for the cell therapies of the future

Science Daily: Pharmacology News

Researchers have developed a new gene switch that can be activated using a commercially available nitroglycerine patch applied to the skin. One day, researchers want to use switches of this kind to trigger cell therapies for various metabolic diseases.

Therapies 259
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Count Me In launches new patient-partnered research project for translocation renal cell carcinoma

Broad Institute

By Count Me In Communications March 11, 2025 Count Me In , a patient-partnered research initiative led by the Broad Institute of MIT and Harvard and Dana-Farber Cancer Institute, is launching a new project focused on translocation renal cell carcinoma (tRCC), a rare and aggressive form of kidney cancer.

Research 113