HOME >> BIOLOGY >> NEWS
University of Pittsburgh reports best gene delivery to date of protein missing in Duchenne Muscular Dystrophy

PITTSBURGH, Nov. 27 Using sophisticated techniques, University of Pittsburgh scientists have engineered perhaps the best gene therapy to date for Duchenne Muscular Dystrophy (DMD), the catastrophic muscle wasting disease that strikes thousands of boys in the United States each year.

The research, conducted in animals and reported in the Nov. 28 issue of the Proceedings of the National Academy of Sciences, is remarkable on several fronts. The Pittsburgh team managed to whittle down the largest gene ever found (14 kilobases) and packaged its most important components (less than 4.2 kilobases) into the smallest and arguably the safest viral vector ever used for gene therapy.

With this work, the investigators demonstrated, for the first time, the minimal amount of the dystrophin gene needed to achieve functional muscle. The gene vector used was a genetically engineered form of adeno-associated virus, or AAV.

When injected into the calf muscles of mice unable to naturally produce the dystrophin protein, the mini-gene construct resulted in the expression of functional dystrophin protein in almost 90 percent of the muscle tissue treated. The dystrophin expression lasted at least one year the duration of the experiments.

Our research showed that we could introduce functional dystrophin into muscle tissue with AAV. This gives us great hope that we can use this gene therapy strategy in a larger animal model of DMD and eventually treat patients within several years, said Xiao Xiao, Ph.D., assistant professor in the department of molecular genetics and biochemistry at the University of Pittsburgh. We were excited to see that mice treated with the mini-dystrophin gene did not show evidence of muscle breakdown many months after treatment, and even after the treated mice exercised, as was seen in untreated mice that lack dystrophin.

With this work, Dr. Xiao and his laboratory have really taken the AAV vector to a new level in its application to the treat
'"/>

Contact: Lauren Ward
wardle@msx.upmc.edu
412-624-2607
University of Pittsburgh Medical Center
27-Nov-2000


Page: 1 2 3 4

Related biology news :

1. Indiana University, EPA to study airborne PCBs
2. University of Alberta researcher looks for clues to mysterious disease
3. Northeastern University receives $12.4 million NSF grant for creation of nanomanufacturing institute
4. Washington University in St. Louis leads group studying aging process
5. Tufts University establishes $4 million dollar tissue engineering resource center
6. Case for IBD combination therapy comes from research at Baylor, MIT and Hebrew University
7. As informatics grows, Indiana University helps set research agenda
8. University of Arizona licenses patent for natural fungicide
9. Washington University in St. Louis plays key role in sequencing moss genome
10. University of Pittsburgh receives $10 million grant for head and neck cancer
11. Clemson University spin-off uses corn to make plastics, provide cleaner air

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:5/14/2020)... ... 13, 2020 , ... DuPont Nutrition & Biosciences (DuPont) today ... dairy market. This unique product allows for process optimization and cost reduction, enabling ... across North America. , Among the numerous benefits Bonlacta™ offers, it is fast ...
(Date:5/1/2020)... ... April 30, 2020 , ... USARAD ... Healthineers and several healthcare VC firms announces an official launch of CareDoctors.com. In ... to enhance patient care experience by offering live video consultations with board certified ...
(Date:4/30/2020)... ... April 28, 2020 , ... Ever ... latest effort in providing timely and critical information to life science organizations as ... and health safety concerns. , “Providing the most up-to-date actionable guidance and information ...
Breaking Biology News(10 mins):
(Date:5/15/2020)... (PRWEB) , ... May 15, 2020 , ... Sentien Biotechnologies, ... appointment of Richard Ganz as Executive Chairman. Mr. Ganz will continue to lead ... as well as work closely with CEO Brian Miller and Sentien’s management team. ...
(Date:5/14/2020)... ... 13, 2020 , ... Early stages of drug discovery often ... information on the drug’s mechanism of action (MOA). Gene expression profiling technologies like ... activity of molecular pathways. This information can complement phenotypic readouts and can be ...
(Date:5/5/2020)... (PRWEB) , ... May 05, 2020 , ... ... solutions company, announced today the launch of its iGlucose® LTE Cat-M ... iGlucose solution utilizes LTE Cat-M1 technology, the industry’s newest and broadest cellular connectivity ...
(Date:4/22/2020)... ... April 20, 2020 , ... Global molecular ... today that Omixon’s founder and chairman Dr. Attila Berces returns as CEO. Outgoing ... HLA since 2014 before stepping into the CEO role in 2017. , ...
Breaking Biology Technology:
Cached News: