Posts tagged gene therapy
Articles and news from the latest research reports.
First gene therapy in Europe
The European Commission confirmed the EMA’s recommendation for market authorisation for the gene therapy Glybera (alipogene tiparvovec), a treatment for patients with lipoprotein lipase deficiency (LPLD) suffering from recurring acute pancreatitis. The rare, inherited disease affects about 350-700 patients in Europe. Patients are unable to metabolise fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis), a potentially lethal condition. Up to now, no gene therapy has been approved in the EU or the US. Glybera consists of an Adeno-associated Virus (AAV) vector expressing the faulty LPL enzyme.
“This therapy will have a dramatic impact on the lives of these patients. Currently their only recourse is to severely restrict the amount of fat they consume”, commented Professor John Kastelein from Academic Medical Center of the University of Amsterdam. “By helping to normalise the metabolism of fat, Glybera prevents inflammation of the pancreas, thereby averting the associated pain and suffering and, if administered early enough, the associated co-morbidities.”
New gene-therapy approach could improve obesity treatment
Medical researchers at the University of Alberta have found a new way of using gene therapy to treat obesity. The treatment was successful, resulting in less weight gain, higher activity levels and decreased insulin resistance in lab models on a high-fat, high-sugar diet.
Faculty of Medicine & Dentistry researcher Jason Dyck, who works in the Department of Pediatrics and the Department of Pharmacology, published his findings this week in the peer-reviewed journal Nutrition and Diabetes. His team found a way to deliver the obesity treatment via DNA as opposed to a virus, which has had limited success in the past, especially over the long term. The results they demonstrated corroborated findings by other researchers who conducted short-term studies or used more risky methods of gene delivery.
“I think our findings may bring this treatment one step closer to clinical trials, as this approach appears to be much safer than conventional forms of gene therapy,” said Dyck.
The obesity treatment focused on increasing levels of adiponectin, a hormone secreted from fat cells. As a person gains weight and fat cells get larger, the body secretes less of this hormone. People who are thin secrete high levels of this hormone.
“This hormone seems to be protective against a number of diseases, including diabetes and cardiovascular disease, as well as weight gain,” says Dyck. “But as you gain weight, less adiponectin is secreted and you lose the beneficial effects associated with this hormone.”
Lab animal models fed a high-fat, high-sugar diet that were given this treatment gained less weight, burned more calories, were more active, used more oxygen, and were better protected against glucose intolerance and insulin resistance than those that were fed the same diet but didn’t get the anti-obesity treatment. Dyck hopes other research teams will move his work forward.
Scientists have restored the sense of smell in mice through gene therapy for the first time — a hopeful sign for people who can’t smell anything from birth or lose it due to disease.
The achievement in curing congenital anosmia — the medical term for lifelong inability to detect odors — may also aid research on other conditions that also stem from problems with the cilia. Those tiny hair-shaped structures on the surfaces of cells throughout the body are involved in many diseases, from the kidneys to the eyes.
The new findings, published online in Nature Medicine, come from a team at the University of Michigan Medical School and their colleagues at several other institutions.
Gene Therapy Treatment Extends Lives of Mice with Fatal Disease, Spinal Muscular Atrophy
July 16th, 2012
Spinal Muscular Atrophy affects one in 6,000 children and has no known cure.
A team of University of Missouri researchers has found that introducing a missing gene into the central nervous system could help extend the lives of patients with Spinal Muscular Atrophy (SMA) – the leading genetic cause of infantile death in the world.
SMA is a rare genetic disease that is inherited by one in 6,000 children who often die young because there is no cure. Children who inherit SMA are missing a gene that produces a protein which directs nerves in the spine to give commands to muscles.
The MU team, led by Christian Lorson, professor in the Department of Veterinary Pathobiology and the Department of Molecular Microbiology and Immunology, introduced the missing gene into mice born with SMA through two different methods: intravenously and directly into the mice’s central nervous systems. While both methods were effective in extending the lives of the mice, Lorson found that introducing the missing gene directly into the central nervous system extended the lives of the mice longer.
Mice born with spinal muscular atrophy typically only live five or six days. Researchers introduced the SMN gene into the mice’s central nervous systems and were able to extend their lives 10-25 days longer. The mice in the picture have spinal muscular atrophy.
“Typically, mice born with SMA only live five or six days, but by introducing the missing SMN gene into the mice’s central nervous systems, we were able to extend their lives 10-25 days longer than SMA mice who go untreated,” said Lorson, who works in the MU Bond Life Sciences Center and the College of Veterinary Medicine. “While this system is still not perfect, what our study did show is that the direct administration of the missing gene into the central nervous system provides some degree of rescue and a profound extension of survival.”
There are several different types of SMA that appear in humans, depending on the age that symptoms begin to appear. Lorson believes that introducing the missing gene through the central nervous system is a way to potentially treat humans regardless of what SMA type they have.
“This is a treatment method that is very close to being a reality for human patients,” Lorson said. “Clinical trials of SMA treatment using gene therapy are likely to begin in next 12-18 months, barring any unforeseen problems.”
Source: Neuroscience News