Gene therapy may be "one shot stop" for rare bone disease
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For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.
"It's been a tremendous success and has proven to be a lifesaving treatment," said José Luis Millán, PhD, professor in the Human Genetics Program at Sanford Burnham Prebys.
Researchers identify genetic 'fingerprint' to predict drug resistance in bacteria
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Antibiotic resistance is a global public health crisis responsible for more than a million deaths annually. By 2050, the World Health Organization estimates it could surpass cancer and heart disease as the leading cause of death as more bacteria develop defenses to the drugs designed to combat them.
Now Tulane University researchers have identified a unique genetic signature in bacteria that can predict their likelihood of developing antibiotic resistance, according to a new study published in Nature Communications.
Harnessing generative AI to treat undruggable diseases
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Biomedical engineers at Duke University have developed an AI-based platform that designs short proteins, termed peptides, capable of binding and destroying previously undruggable disease-causing proteins. Inspired by OpenAI’s image generation model, their new algorithm can rapidly prioritize peptides for experimental testing.
The work appeared Jan. 22 in the journal Science Advances.
Microbial therapy offers new hope for vitiligo patients
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A natural compound derived from gut-friendly bacteria significantly slows the progression of vitiligo and may restore pigmentation, reports a new Northwestern University pre-clinical study in mice.
The findings could offer hope to millions affected by the autoimmune disease, which causes visible patches of skin discoloration and carries profound emotional and physical consequences.
Skin cancer: New treatment option successfully tested
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Basal cell carcinomas, the most common form of skin cancer, occur in chronically sun-exposed areas such as the face. Locally advanced tumours in particular can be difficult to treat surgically. A research team from MedUni Vienna and University Hospital Vienna has now investigated the effectiveness of a new type of therapy and achieved promising results: The active substance TVEC led to a reduction in the size of the basal cell carcinoma in all study participants, which not only improved surgical removal, but also led to a complete regression of the tumour in some of the patients.
AI paves the way for personalised treatments in ageing science
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A collaborative study between researchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine), and the Institute for Biostatistics and Informatics in Medicine and Ageing Research, Rostock University Medical Center, Germany, investigated how advanced AI tools, like Large Language Models (LLMs), can make it easier to evaluate interventions for ageing and provide personalised recommendations. The findings were published in the leading review journal Ageing Research Reviews.
Scientists design peptides to enhance drug efficacy
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A team of scientists has developed a groundbreaking approach using specially designed peptides to improve drug formulations. This innovative method significantly enhances anti-tumor efficacy, as demonstrated in leukemia models. The study, published in the journal Chem, was led by researchers at the Advanced Science Research Center at the CUNY Graduate Center (CUNY ASRC) and Memorial Sloan Kettering Cancer Center.
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