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 […]

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 […]

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 […]

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 […]

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 […]

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 […]