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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 312
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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 323
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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 310
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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 279
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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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With new IPO pitch, Artiva touts ‘natural killer’ cell therapy for autoimmune disease

BioPharma Drive: Drug Pricing

Like many of its cell therapy peers, Artiva, which originally sought an IPO in 2021, has shifted its strategy in hopes of riding a recent wave of investor interest in autoimmune disease research.

Therapies 193
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Engineers turn skin cells directly into neurons for cell therapy

Science Daily: Pharmacology News

Researchers devised a process to convert a skin cell directly into a neuron, eliminating the need to generate induced pluripotent stem cells. Such neurons could be used to treat spinal cord injuries or diseases such as ALS.