HOME >> BIOLOGY >> NEWS
With government nod, gene therapy pioneered at UNC is given to children with rare disorder

CHAPEL HILL -- A form of gene therapy created and produced at the University of North Carolina at Chapel Hill and approved by the U.S. Food and Drug Administration has been given to children with a rare, inherited neurological disorder.

This work involved collaboration with investigators at the Robert Wood Johnson Medical School, of the University of Medicine and Dentistry of New Jersey, and was led by Dr. Paola Leone and a group of 16 investigators and surgeons.

The children have Canavan disease, characterized by spongy degeneration of the brain's white matter. The disease affects the growth of the fatty myelin sheath insulating nerve fibers.

Canavan disease symptoms appear in early infancy and progress rapidly. These may include mental retardation, loss of previously acquired motor skills, feeding difficulties, abnormal muscle tone (floppiness or stiffness), poor head control and megalocephaly (abnormally enlarged head). Paralysis, blindness or hearing loss also may occur.

There is no cure for Canavan disease or a standard course of treatment other than symptomatic and supportive. Prognosis is poor; victims usually die before age 10.

"Children with Canavan disease have a mutation in one of their genes, causing a deficiency of an enzyme responsible for removing cellular waste," said Dr. R. Jude Samulski, professor of pharmacology at UNC's School of Medicine and director of the Gene Therapy Center. "In this disease, waste product builds up, causing brain cells to die."

Absolutely nothing can be done to prevent disease progression, said Samulski, which is why the objective was to place a gene into the brain that would express the missing enzyme.

The therapy received by the children uses a genetically altered adeno-associated virus, or AAV, to deliver genes into the body. Samulski, a pioneer in AAV research, said he chose to study and develop altered AAV for several reasons, including its potential for fewer toxic effects than that
'"/>

Contact: Leslie Lang
llang@med.unc.edu
919-843-9687
University of North Carolina School of Medicine
30-May-2003


Page: 1 2 3

Related biology news :

1. Landmark gene agreement announced by Samoan government and UC Berkeley for anti-AIDS drug Prostratin
2. East Asian governments urged to harness market forces to improve environment, save money
3. Patagonias coastal wildlife receive a protective boost by Argentine government
4. Diet a major factor in breast cancer time for government action
5. Local, regional governments could take lead in curbing global air pollution
6. CIESIN receives grant to establish government biological information site
7. Study says government counts of tigers in India are inaccurate
8. Anthrax threat needs aggressive government action plan, say researchers
9. U of A scientist finds Atlantic salmon escapes higher than government reports
10. Sandia-aided method to heal wounded and diseased achieves US government acceptance
11. Public calls on U.S. government to protect the worlds coral reefs, new poll finds

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


(Date:8/3/2020)... ... August 03, 2020 , ... Accumen Inc. is helping healthcare ... manufacturer, Spectrum Solutions, to provide COVID-19 saliva testing kits nationally. , “Accumen ... more than 10 years. Early in the COVID-19 crisis, we identified supply chain ...
(Date:7/22/2020)... ... 21, 2020 , ... USDM Life Sciences , a ... a new solution to manage regulated workloads on Microsoft Azure. , Regulated biotechnology, ... complies with FDA and global regulations. USDM's new managed service for regulated workloads ...
(Date:7/10/2020)... ... July 08, 2020 , ... Overcoming Comparability Issues in Regenerative ... Webinar, Wednesday, July 22, 2020 • 1:30 p.m.-3:00 p.m. EDT, https://www.fdanews.com/cellandgenetherapy ... effective way to complete one? Will the study comply with all FDA requirements? ...
Breaking Biology News(10 mins):
(Date:8/3/2020)... (PRWEB) , ... August 03, 2020 , ... ... collection, today announced Jim Corrigan, President and CEO has been named one of ... broad cross-section of industry sectors, PharmaVoice 100 honorees are selected based on how ...
(Date:7/31/2020)... ... July 29, 2020 , ... ... transmitter systems that can be configured to drive Klystrons, TWTs, IOTs, and magnetrons. ... drive one or two switches in a push-pull configuration; yielding fast fall time ...
(Date:7/31/2020)... ... 2020 , ... The SDX® Respiratory Gating System , ... reached its 20th anniversary of worldwide use. Introduced in the US over the ... University of Pennsylvania, University of Michigan, University of Maryland, University of California San ...
(Date:7/18/2020)... ... 2020 , ... “We are thrilled to deliver this new technology to the ... its kind on the market and we were pleased that the IFT jury recognized ... cultured ingredients, creating a natural way to extend the shelf life and improve the ...
Breaking Biology Technology:
Cached News: