Phase I/II clinical study of gene therapy for GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases, shows encouraging results
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- Category: Research
Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the disease with minimal adverse reactions.
"Biochemically it worked," said study investigator Heather Gray-Edwards, DVM, PhD, assistant professor of genetic & cellular medicine at UMass Chan.
New discovery could lower heart attack and stroke risk for people with type 2 diabetes
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New research from the Charles Perkins Centre at the University of Sydney has uncovered a new biological pathway that may help explain why people with type 2 diabetes are more prone to developing dangerous blood clots, potentially paving the way for future treatments that reduce their cardiovascular risk.
The study, led by Associate Professor Freda Passam from the Central Clinical School and Associate Professor Mark Larance from the School of Medical Sciences, was published in the Journal of Clinical Investigation.
ADAMTS2: A multifunctional protein with therapeutic potential
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- Category: Research
ADAMTS2, a member of the ADAMTS zinc metalloproteinase family, is widely recognized for its pivotal role as a procollagen I N-proteinase. This enzyme plays a crucial part in the maturation of fibrillar collagens, which are essential for maintaining the structural integrity of connective tissues. Beyond its traditional role, recent discoveries have revealed that ADAMTS2 is involved in a diverse range of biological processes that extend well beyond collagen maturation.
AI revolution in medicine: how large language models are transforming drug development
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- Category: Research
The pharmaceutical industry stands at a transformative crossroads as artificial intelligence reshapes the landscape of drug development. In a Correspondence published in the KeAi journal Current Molecular Pharmacology, a group of researchers from China illuminate how large language models (LLMs) - the sophisticated AI systems powering advanced chatbots - are delivering unprecedented breakthroughs across the entire drug discovery pipeline.
Cancer drug eliminates aggressive cancers in clinical trial
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- Category: Research
Over the past 20 years, a class of cancer drugs called CD40 agonist antibodies have shown great promise - and induced great disappointment. While effective at activating the immune system to kill cancer cells in animal models, the drugs had limited impact on patients in clinical trials and caused dangerously systemic inflammatory responses, low platelet counts, and liver toxicity, among other adverse reactions - even at a low dose.
A patch for the heart
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- Category: Research
Following a heart attack, blood flow to the heart is interrupted and the resulting lack of oxygen can cause heart damage. The heart wall can rupture in severe cases, requiring immediate surgical intervention. Today, bovine pericardial patches are used to repair such heart defects owing to their stability, permeability, and ease of implantation.
The meditation app revolution is here, and it's backed by science
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- Category: Research
Do you have a meditation app on your smartphone, computer or wearable device? Well, you're not alone.
There are now thousands of meditation apps available worldwide, the top 10 of which have been collectively downloaded more than 300 million times. What’s more, early work on these digital meditation platforms shows that even relatively brief usage can lead to benefits, from reduced depression, anxiety, and stress to improved insomnia symptoms.
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