Longevity science

Researchers said on Tuesday they planned to enroll patients into two new clinical trials using Mydicar, a gene therapy treatment made by privately held U.S. biotech company Celladon.

After more than 20 years of research, the ground-breaking method for fixing faulty genes is starting to deliver, with European authorities approving the first gene therapy for an rare metabolic disease last November.

In the case of heart failure, the aim is to insert a gene called SERCA2a directly into heart cells using a modified virus, delivered via a catheter infusion. Lack of SERCA2a leads to ever weaker pumping in people with heart failure.

Researchers from the Universitat Autònoma de Barcelona (UAB) have claimed a first by successfully using a single session of gene therapy to cure dogs of type 1 diabetes. The work has shown that it is possible to cure the disease in large animals with a minimally-invasive procedure – potentially leading the way to further developments in studies for human treatment of the disease.

From cancer treatments to new devices to gene therapy, a look at six medical innovations that are poised to transform the way we fight disease.

The latest in the fight against ever dying is a gene therapy that gives mice a healthy dose of telomerase, the enzyme that keeps our chromosomes – and thus our cells and bodies – “young.”

The therapy extended the lifespans of mice by 24 percent and, at least so far, the therapy appears to be completely safe...

Researchers at Northeastern University in Boston have developed a gene therapy approach that may one day stop Parkinson’s disease (PD) in it tracks, preventing disease progression and reversing its symptoms.

The novelty of the approach lies in the nasal route of administration and nanoparticles containing a gene capable of rescuing dying neurons in the brain.

One of the biggest problems in treating HIV patients is the amount of daily individual medications it takes to keep the virus at bay. In a new study, scientists at the Stanford University School of Medicine have engineered a new approach to tailored gene therapy that they say makes key cells of the immune system resistant to attack from the HIV virus, which may eventually lead to the removal of life-long dependencies on drugs for patients living with HIV.

A clinical trial testing a gene therapy for HIV patients is now 11 years old. Recently, the researchers running the study published an examination of the patients after all this time.

Of the study’s 43 patients, all were healthy, and 41 of them confirmed that their immune cells which received a genetically-altered boost were still performing as hoped more than a decade after the initial infusions...