The success of a gene therapy for blindness caused by a genetic mutation paves the way for gene therapies which treat other forms of blindness as well as similar treatments which treat other diseases.
FDA approves novel gene therapy to treat patients with a rare form of blindness. The first gene therapy approved for inherited disease.
In a paradigm shift, scientists are starting to regard respiratory disease as a disease of aging and scientists at the Mayo Clinic reversed symptoms of a lung disease in mice using age-reversing senolytic compounds. In addition to senolytics, researchers are developing other treatments for respiratory conditions such as gene therapy.
Summary: In a paradigm shift, scientists are starting to regard respiratory disease as a disease of aging and scientists at the Mayo Clinic reversed symptoms of a lung disease in mice using age-reversing senolytic compounds. In addition to senolytics, researchers are developing other treatments for respiratory conditions such as gene therapy. [This article first appeared on the website LongevityFacts.com. Author: Brady Hartman.]
All of us – both smokers and non-smokers alike – will lose significant lung capacity as we age. Scientists at the Mayo Clinic have developed a promising treatment that rejuvenates the damaged lungs of mice using anti-aging compounds called senolytics.
Continue reading “Breakthrough Cures for Respiratory Diseases and COPD in the Pipeline” »
Elizabeth Parrish is a proponent of controversial ideas. Rankled by barriers to trials on potential life-enhancing treatments, she used herself as a guinea pig and says the results have borne fruit – but she has irked the science community in the process.
Experimenting on herself didn’t go down well with scientists, but Elizabeth Parrish is convinced gene therapy can increase the length and quality of our lives, and wants early approval for treatments to stem ‘planet’s biggest killer’.
Some 50,000 people in the U.S. are diagnosed with Parkinson’s disease (PD) every year. The American Institute of Neurology estimates there are one million people affected with this neurodegenerative disorder, with 60 years as average age of onset. Falls and fall-related injuries are a major issue for people with Parkinson’s?up to 70 percent of advanced PD patients fall at least once a year and two-thirds suffer recurring falls. These fall rates are twice as high as those of adults of comparable age, so improving balance in patients with Parkinson’s would provide a major health advantage.
Sunil Agrawal, professor of mechanical engineering and of rehabilitation and regenerative medicine at Columbia Engineering, along with Dario Martelli, a post-doctoral researcher in his group, have been working on this issue with Movement Disorders faculty from the department of neurology at Columbia University Medical Center?Stanley Fahn, a leading expert in Parkinson’s, and Un Jung Kang, division director, and Movement Disorder Fellow Lan Luo. In their latest study, published today in Scientific Reports, the team looked at whether or not Parkinson’s disease affects patients’ balance and diminishes their ability to react and adapt to walking with perturbations. The researchers found that the ability to adapt to multiple perturbations or to modify responses to changing amplitudes or directions was not affected by PD; both the Parkinson’s and the healthy subjects controlled their reactive strategies in the same way.
A single infusion gene therapy treatment improved levels of the essential blood clotting protein Factor VIII, with 85 percent of patients achieving normal or near-normal levels of the blood clotting factor, even many months after treatment.
Summary: British doctors say they have achieved “mind-blowing” results using gene therapy to rid people of hemophilia A. [This article first appeared on LongevityFacts. Author: Brady Hartman.]
We are one step closer to a cure for hemophilia according to the results of a groundbreaking gene therapy trial published in the New England Journal of Medicine.
Continue reading “Groundbreaking Gene Therapy Trial Aims to Cure Hemophilia” »
New view on mitochondrial DNA could put the brakes on mutations that drive diseases. Scientists perform landmark sequencing of mitochondrial DNA and discover surprising facts.
Summary: New view on mitochondrial DNA could help put the brakes on mutations that drive diseases. [Author: Brady Hartman. This article first appeared on LongevityFacts.]
DNA sequences between mitochondria inside a single cell are vastly different, reported scientists in the Perelman School of Medicine at the University of Pennsylvania. This discovery will help to illuminate the underlying mechanisms of diseases that start with mutations in mitochondrial DNA and provide clues about how patients might respond to specific treatments. The researchers published their findings in the journal Cell Reports this week.
Continue reading “Researchers Discover Key to Diseases in Mitochondrial DNA Mutations” »
Gene therapy is the process of replacing defective genes with healthy ones, adding new genes to help the body fight or treat disease, or deactivating problem genes. It holds the promise to transform medicine and create options for patients who are living with difficult, and even incurable, diseases. Learn how this innovative therapy works.
Continuous automatic sampling during production aims to keep Danish biotechnology at the forefront. The equipment, and the company behind it, are the result of fruitful collaboration between businesses and universities.
Biotech companies can now take samples from their production as often as they wish, untouched by human hands.
This is all thanks to new equipment developed by start-up company Biomatics Technology. Both the company and product were nurtured in the Biopro network, which involves a number of Danish biotech companies and DTU and the University of Copenhagen.
