Experimental gene therapy for giant axonal neuropathy shows promise in NIH clinical trial
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An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health (NIH). Currently, there is no treatment for GAN and the disease is usually fatal by 30 years of age. Fourteen children with GAN, ages 6 to 14 years, were treated with gene transfer therapy at the NIH Clinical Center and then followed for about six years to assess safety.
Cell therapy approach harnesses the immune system in a different way to stop cancer
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A new cancer treatment that uses a person’s own immune cells has been approved by the U.S. Food and Drug Administration (FDA) for treating the most dangerous type of skin cancer. Now this form of cellular therapy (tumor-infiltrating lymphocyte - or TIL therapy) is showing promise in advanced lung cancers through clinical trials underway at The Ohio State University Comprehensive Cancer Center - Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC - James).
Bacteria commonly found in the body contribute to stomach cancer
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A new study has discovered that a type of bacteria commonly found in the body, which usually does not pose problems for healthy people, plays a significant role in causing stomach cancer, the fifth most common cancer in the world.
Streptococcus anginosus bacteria exist alongside other germs in the mouth, throat, intestines and vagina. Occasionally, they may cause mild infections like sore throats and skin infections.
Novel molecules from generative AI to Phase II
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There are thousands of diseases worldwide with no cure or available treatments. Traditional drug discovery and development takes decades and billions of dollars and more than 90% of these drugs fail in clinical trials. The emergence of artificial intelligence (AI) holds promise for streamlining and improving the entire process. However, ushering in a new era of AI-driven drug discovery requires costly and lengthy validation in preclinical cell, tissue, and animal models and human clinical trials.
Key protein linked to immune disorders
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A new study has shed light on the importance of the protein STAP-1 in activating certain immune cells. Understanding the role of STAP-1 in these cells could give researchers a better glimpse into immune-related disorders and ways to treat them.
The researchers found that STAP-1 plays an important role in the activation of T cells, which are white blood cells that play a critical role in defending the body against infections and maintaining overall health.
Researchers open new leads in anti-HIV drug development, using a compound found in nature
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A team of University of Michigan researchers has successfully modified a naturally occurring chemical compound in the lab, resulting in advanced lead compounds with anti-HIV activity.
Their results, published March 7 in the Journal of Medicinal Chemistry, offer a new path forward in the development of drugs that could potentially help cure - rather than treat - HIV.
Researchers discover new cancer-fighting role for neutrophils
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In a study published in Cell on March 5, Prof. ZHANG Xiaoming at the Shanghai Institute of Immunity and Infection (SIII) of the Chinese Academy of Sciences and Profs. GAO Qiang, FAN Jia and YANG Li at Fudan University have uncovered an unexpected level of complexity hidden within neutrophils, which were previously thought to be a relatively uniform population of short-lived immune cells.
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