Personalized Medicine

The promise of personalized medicine is not for everyone

Could your medical treatment one day be tailored to your DNA? That’s the promise of “personalized medicine,” an individualized approach that has caught the imagination of doctors and researchers over the past few years. This concept is based on the idea that small genetic differences between one person and another can be used to design tailored treatments for conditions as diverse as cancer and schizophrenia. In principle, “personalized” is not
Gene Therapy

New gene therapy corrects a form of inherited macular degeneration in canine model

Researchers from the University of Pennsylvania have developed a gene therapy that successfully treats a form of macular degeneration in a canine model. The work sets the stage for translating the findings into a human therapy for an inherited disease that results in a progressive loss of central vision and which is currently untreatable. The study, published today in Proceedings of the National Academy of Sciences, was led by Karina
cancer Gene Therapy Genomes Hepatitis

New Treatment Leaves Liver Cancer Cells In Limbo

Scientists have shown that a mutation in a gene called Arid1b can cause liver cancer. The gene normally protects against cancer by limiting cell growth, but when mutated it allows cells to grow uncontrollably. The researchers have shown that two existing drugs can halt this growth in human cells. This points to a new approach to treating liver cancer. These early results could be translated into a treatment relatively quickly,
Clinical Trials Gene Therapy Vaccines

Smallest-Reported Artificial Virus Could Help Advance Gene Therapy

Gene therapy is a kind of experimental treatment that is designed to fix faulty genetic material and help a patient fight off or recover from a disease. Now scientists have engineered the smallest-reported virus-like shell that can self-assemble. It could someday carry potentially therapeutic DNA or RNA and transfer it to human cells. The report appears in the Journal of the American Chemical Society. The story of gene therapy is
Alzheimers and Dementia Genomes Neurodegenerative diseases Neurology

Genetic ‘Switch’ Identified As Potential Target For Alzheimer’S Disease

A team at the MRC Clinical Sciences Centre (CSC), based at Imperial College London, has found an important part of the machinery that switches on a gene known to protect against Alzheimer’s Disease. Working in collaboration with scientists at the Hong Kong University (HKU) and the Erasmus University in Rotterdam, CSC associate professor Richard Festenstein explored the steps by which this Neuroglobin gene is gradually switched on, or up-regulated. Neuroglobin
rare diseases

NIH researchers discover otulipenia, a new inflammatory disease

National Institutes of Health researchers have discovered a rare and sometimes lethal inflammatory disease - otulipenia - that primarily affects young children. They have also identified anti-inflammatory treatments that ease some of the patients' symptoms: fever, skin rashes, diarrhea, joint pain and overall failure to grow or thrive. Otulipenia is caused by the malfunction of OTULIN, a single gene on chromosome 5. When functioning properly, OTULIN regulates the development of
Gene Therapy rare diseases

New Collaboration focuses on Gene Therapy to Tackle Epidermolysis Bullosa (EB) Treatments

Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on delivering gene and plasma-based therapy for life-threatening rare diseases, EB Research Partnership (EBRP) and EB Research Medical Foundation (EBMRF) announced today a collaboration focusing on gene therapy treatments for epidermolysis bullosa (EB), a group of devastating rare genetic skin disorders impacting children; characterized by skin blisters and erosions all over the body. Phase 1 clinical trial results for the lead EB
cancer Gene Therapy Genomes

Restoring Chemotherapy Sensitivity by Boosting MicroRNA Levels

By increasing the level of a specific microRNA (miRNA) molecule, researchers have for the first time restored chemotherapy sensitivity in vitro to a line of human pancreatic cancer cells that had developed resistance to a common treatment drug. If the miRNA molecules can be delivered to cells in the human body – potentially with nanoparticles – the technique might one day be used to battle the chemotherapy resistance that often
Gene Therapy Genomes

TSRI Team Streamlines Biomedical Research by Making Genetic Data Easier to Search

Call them professional “data wranglers.” A team of scientists at The Scripps Research Institute (TSRI) is expanding web services to make biomedical research more efficient. With their free, public projects, MyGene.info and MyVariant.info, researchers around the world have a faster way to spot new connections between genes and disease. “This is about how to deliver information quickly to biologists,” said Chunlei Wu, associate professor of molecular medicine at TSRI. Wu