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Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment

Science Daily: Pharmacology News

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.

Treatment 321
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Researchers make breakthrough in bioprinting functional human heart tissue

Science Daily: Pharmacology News

The breakthrough science focused on replicating heart tissues, bringing research closer to generating functional, bioprinted organs, which would have broad applications in disease modelling, drug screening and regenerative medicine.

Research 323
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David Liu receives Breakthrough Prize in Life Sciences

Broad Institute

David Liu receives Breakthrough Prize in Life Sciences By Corie Lok April 5, 2025 Breadcrumb Home David Liu receives Breakthrough Prize in Life Sciences Liu is honored for the development of base editing and prime editing, two gene editing technologies transforming medicine. In 2013, Broad founding director Eric S.

Science 144
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Parkinson's Disease: New theory on the disease's origins and spread

Science Daily: Pharmacology News

New hypothesis paper builds on a growing scientific consensus that Parkinson's disease route to the brain starts in either the nose or the gut and proposes that environmental toxicants are the likely source.

Disease 363
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Science Shows How Night Shifts Help Bring on Disease

Drugs.com

TUESDAY, May 14, 2024 -- Night shift work can increase a person’s risk of chronic disease, and a new study reveals one possible explanation for this.It appears that just a few days on a night shift schedule throws off body rhythms tied to.

Disease 246
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Sweat health monitor measures levels of disease markers

Science Daily: Pharmacology News

The 3D-printed monitor could someday provide a simple and non-invasive way to track health conditions and diagnose common diseases, such as diabetes, gout, kidney disease or heart disease. A wearable health monitor can reliably measure levels of important biochemicals in sweat during physical exercise.

Disease 329
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Alcohol raises heart disease risk, particularly among women

Science Daily: Pharmacology News

Young to middle-aged women who reported drinking eight or more alcoholic beverages per week--more than one per day, on average--were significantly more likely to develop coronary heart disease compared with those who drank less, finds a study presented at the American College of Cardiology's Annual Scientific Session.

Disease 351