Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Clinical trial for muscular dystrophy demonstrates safety of customized gene therapy

01.12.2011
Researchers at the University of North Carolina at Chapel Hill have shown that it is safe to cut and paste together different viruses in an effort to create the ultimate vehicle for gene therapy. In a phase I clinical trial, the investigators found no side effects from using a "chimeric" virus to deliver replacement genes for an essential muscle protein in patients with muscular dystrophy.

"This trial demonstrates that gene therapy is no longer limited by the viruses we find in nature, and should usher in the next generation of viral delivery systems for human gene transfer," said senior study author R. Jude Samulski, PhD, professor of pharmacology and director of the Gene Therapy Center at UNC. The study appears online in the Nov. 8, 2011 issue of the journal Molecular Therapy.

Through gene therapy, scientists treat diseases by correcting a patient's faulty genes. Most of the time, this approach involves commandeering a natural system for infecting and introducing new genes into cells; thus, the virus. But even though there are lots of relatively innocuous viruses available for this purpose, none of them are perfectly suited for gene therapy.

Rather than rely on nature, Samulski and his colleagues decided to engineer their dream gene therapy virus in the laboratory. First they chose the adeno-associated virus or AAV, a small nonpathogenic virus that most humans are exposed to at some point in life. They then took their favorite attributes from different forms of AAV – such as AAV type 1's ability to sneak into muscle, and AAV type 2's safe track record – and combined them into one "chimeric" virus. In the first trial of this form of gene therapy, the investigators gave six boys with Duchenne muscular dystrophy (DMD) this new virus. An x-linked inherited disorder, DMD affects one in 4,000 newborn boys.

The virus was engineered to contain the dystrophin gene, which is missing in patients with muscular dystrophy and is the ultimate cause of the disease's progressive muscle weakness. The replacement genes were injected into the bicep in one arm and a placebo was injected into the other arm of each of the patients. The researchers were able to detect the new genes in all of the patients treated with the gene therapy, but no immunological response.

As they move on to the next phase of clinical trials, Samulski says they are carefully considering how best to administer the gene therapy vectors to patients. Delivering enough replacement genes to a therapeutic effect could require larger doses of virus, which in turn could elicit an unwanted immune response. So the researchers are exploring a number of different options, including using a new high pressure technique developed by William J. Powers, MD, professor and chair of neurology at UNC, reported last July in the same journal, to get the virus into muscle at lower doses.

Study co-authors from UNC include Dawn E. Bowles, PhD; Scott W.J. McPhee, PhD, MPH; Chengwen Li, PhD; Steven J. Gray, PhD; Jade J. Samulski, Angelique S. Camp, Juan Li, MD; Bing Wang, Paul E. Monahan, MD; Joshua C. Grieger, PhD; and Xiao Xiao, PhD.

The UNC research was funded by the National Institutes of Health, the Muscular Dystrophy Association and a grant from the Senator Paul D. Wellstone Muscular Dystropy Cooperative Research Center funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases.

Les Lang | EurekAlert!
Further information:
http://www.unc.edu

More articles from Health and Medicine:

nachricht New 3D cultured cells mimic the progress of NASH
02.04.2020 | Tokyo University of Agriculture and Technology

nachricht Geneticists are bringing personal medicine closer to recently admixed individuals
02.04.2020 | Estonian Research Council

All articles from Health and Medicine >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Harnessing the rain for hydrovoltaics

Drops of water falling on or sliding over surfaces may leave behind traces of electrical charge, causing the drops to charge themselves. Scientists at the Max Planck Institute for Polymer Research (MPI-P) in Mainz have now begun a detailed investigation into this phenomenon that accompanies us in every-day life. They developed a method to quantify the charge generation and additionally created a theoretical model to aid understanding. According to the scientists, the observed effect could be a source of generated power and an important building block for understanding frictional electricity.

Water drops sliding over non-conducting surfaces can be found everywhere in our lives: From the dripping of a coffee machine, to a rinse in the shower, to an...

Im Focus: A sensational discovery: Traces of rainforests in West Antarctica

90 million-year-old forest soil provides unexpected evidence for exceptionally warm climate near the South Pole in the Cretaceous

An international team of researchers led by geoscientists from the Alfred Wegener Institute, Helmholtz Centre for Polar and Marine Research (AWI) have now...

Im Focus: Blocking the Iron Transport Could Stop Tuberculosis

The bacteria that cause tuberculosis need iron to survive. Researchers at the University of Zurich have now solved the first detailed structure of the transport protein responsible for the iron supply. When the iron transport into the bacteria is inhibited, the pathogen can no longer grow. This opens novel ways to develop targeted tuberculosis drugs.

One of the most devastating pathogens that lives inside human cells is Mycobacterium tuberculosis, the bacillus that causes tuberculosis. According to the...

Im Focus: Physicist from Hannover Develops New Photon Source for Tap-proof Communication

An international team with the participation of Prof. Dr. Michael Kues from the Cluster of Excellence PhoenixD at Leibniz University Hannover has developed a new method for generating quantum-entangled photons in a spectral range of light that was previously inaccessible. The discovery can make the encryption of satellite-based communications much more secure in the future.

A 15-member research team from the UK, Germany and Japan has developed a new method for generating and detecting quantum-entangled photons at a wavelength of...

Im Focus: Junior scientists at the University of Rostock invent a funnel for light

Together with their colleagues from the University of Würzburg, physicists from the group of Professor Alexander Szameit at the University of Rostock have devised a “funnel” for photons. Their discovery was recently published in the renowned journal Science and holds great promise for novel ultra-sensitive detectors as well as innovative applications in telecommunications and information processing.

The quantum-optical properties of light and its interaction with matter has fascinated the Rostock professor Alexander Szameit since College.

All Focus news of the innovation-report >>>

Anzeige

Anzeige

VideoLinks
Industry & Economy
Event News

13th AKL – International Laser Technology Congress: May 4–6, 2022 in Aachen – Laser Technology Live already this year!

02.04.2020 | Event News

“4th Hybrid Materials and Structures 2020” takes place over the internet

26.03.2020 | Event News

Most significant international Learning Analytics conference will take place – fully online

23.03.2020 | Event News

 
Latest News

Capturing 3D microstructures in real time

03.04.2020 | Materials Sciences

First SARS-CoV-2 genomes in Austria openly available

03.04.2020 | Life Sciences

Do urban fish exhibit impaired sleep? Light pollution suppresses melatonin production in European perch

03.04.2020 | Life Sciences

VideoLinks
Science & Research
Overview of more VideoLinks >>>