Results from a 20-year study by researchers in the Department of Genetic Medicine at Weill Cornell Medicine show gene therapy's potential to significantly extend survival in children with CLN2 disease, a rare neurodegenerative disorder.
With the 25th anniversary of the Belfer Gene Therapy Core Facility approaching next year, Dr. Stephen Kaminsky, professor of research in genetic medicine and associate director of the facility, shared with us the history and successes of this cutting-edge research facility.
One of the hardest points on the translational road “from bench to bedside” can be the point where you have to turn over your discovery to a company you’ve founded—a company whose subsequent direction you won’t fully control.
Custom-made versions of a widely prescribed, low-cost drug used to prevent a leading cause of blindness in the elderly vary widely in their dosages, Weill Cornell Medical College scientists found in analyses of the eye injections. Investigators saw no evidence of impurities or contamination but found that many of the samples, which were prepared by pharmacists through a process called compounding, contained less medication than doses of the drug obtained directly from the manufacturer. The research, published today in JAMA Ophthalmology, is likely to increase scrutiny of compounding pharmacies, which tailor-mix drugs for individual patients, and to question whether eye-care specialists should continue to prescribe the cancer drug Avastin for age-related macular degeneration (AMD).
A team led by collaborating researchers from Weill Cornell Medicine, Weill Cornell Medicine—Qatar and Qatar Foundation have assembled a large genomic database on the Qatari people, and have used it to develop an advanced but low-cost screening tool for genetic diseases in this highly distinct Middle Eastern population.
Dr. Ronald Crystal, chairman of the Department of Genetic Medicine and the Bruce Webster Professor of Internal Medicine at Weill Cornell Medicine, has been elected a fellow of the National Academy of Inventors (NAI).
The Office of Academic Integration (OVPAI) has awarded $750,000 in seed grants to 10 studies ranging from refugee health and legal rights, to a vaccine treating fentanyl addiction and overdose, to pancreatic cancer and antibiotic tolerance.
A gene therapy developed by Weill Cornell Medicine and NewYork-Presbyterian investigators helped slow progression of a rare and fatal genetic disorder in children called late infantile Batten disease, in a phase 1 clinical study.
There’s a rare hereditary disorder of the connective tissue that causes abnormal twists and turns in the blood vessels. This condition, known as arterial tortuosity syndrome (ATS), can lead to serious aortic complications—including narrowing (stenosis), bulging (aneurysms) and tearing (dissection) of the wall—and can be fatal in early childhood, although individuals with milder forms of the disease can live into adulthood.
A rapid, non-invasive eye exam that uses innovative imaging technology effectively measures the severity of disease in patients with a rare neurodegenerative disease called Friedrich ataxia, according to a study by Weill Cornell Medicine, Weill Cornell Medicine-Qatar and NewYork-Presbyterian researchers. The results suggest that the exam, known as corneal confocal microscopy (CCM), could be a rapid and sensitive tool for assessing patients in the clinic and act as a biomarker in clinical trials testing new therapies for the disease.
A rapid, non-invasive eye exam that uses innovative imaging technology effectively measures the severity of disease in patients with a rare neurodegenerative disease called Friedrich ataxia, according to a study by Weill Cornell Medicine, Weill Cornell Medicine-Qatar and NewYork-Presbyterian researchers. The results suggest that the exam, known as corneal confocal microscopy (CCM), could be a rapid and sensitive tool for assessing patients in the clinic and act as a biomarker in clinical trials testing new therapies for the disease.
Levels of air pollution defined as “good” by the Environmental Protection Agency (EPA) may still harm the lungs of cigarette smokers, according to a new study conducted by Weill Cornell Medicine and NewYork-Presbyterian investigators.
Up to 30 percent of HIV patients who are appropriately treated with antiretroviral therapies develop the chronic lung disease emphysema in their lifetime. Now, new research from Weill Cornell Medicine investigators has uncovered a mechanism that might explain why this lung damage occurs.
Seven winners have been selected for the third round of the Daedalus Fund for Innovation awards, an innovative Weill Cornell Medicine program that helps advance promising applied and translational research projects and emerging technologies that have commercial potential. The projects are wide ranging, designed to address patient needs in areas ranging from newborn medicine to cancer and infectious disease, brain injury and chronic illness.
A vaccine developed at Weill Cornell Medicine and NewYork-Presbyterian to blunt the effects of cocaine has advanced to clinical trials for testing in humans. After demonstrating that the vaccine prevented cocaine from reaching the brain in earlier animal studies, investigators are now enrolling active cocaine addicts in a Phase I randomized control study to test how it works in people.
Researchers at Weill Cornell Medical College have successfully tested their novel anti-cocaine vaccine in primates, bringing them closer to launching human clinical trials.
Researchers have produced a lasting anti-cocaine immunity in mice by giving them a safe vaccine that combines bits of the common cold virus with a particle that mimics cocaine.
