Bay Area Forum for Asians

	Nutopia Forum Index

Nutopia Forum Index

Free Medical Info

Gene Therapy May Help Muscular Dystrophy Patients

View previous topic :: View next topic

Page 1 of 1

	Gene Therapy May Help Muscular Dystrophy Patients Tue Jul 26, 2011 2:54 am

Author

Message

DoctorNutopia

萬人迷

Joined: 06 Nov 2009

Posts: 2981

Post subject: Gene Therapy May Help Muscular Dystrophy Patients

Gene Therapy May Help Muscular Dystrophy Patients

July 25, 2011 -- Scientists say they have successfully tested a new treatment that may one day help children with a severe form of muscular dystrophy.

Duchenne muscular dystrophy is caused by the absence of a protein that helps keep muscle cells intact. It affects about one in every 3,500 newborns and causes progressive muscle weakness and early death.

Children with the disease, who are usually boys, typically lose the ability to walk by age 12 and often die before age 25, usually from heart and lung problems.

Treatments aim to slow the gradual decline and manage symptoms. There's no cure.

Fixing a Flawed Gene The new therapy uses a clever bit of genetic sleight-of-hand to repair an inherited defect in the blueprints the body uses to make the muscle protein dystrophin.

The treatment uses artificially created nucleotides, the building blocks of DNA and RNA, to mask the defect, helping cells to skip over the flawed part of the protein's instructions.

The end result is a shortened but functional protein.

To understand what the treatment does, Andrian R. Krainer, PhD, a professor molecular genetics at Cold Spring Harbor Laboratory in New York, says it helps to think of the protein as a long spring.

"If it's missing one of the ends, it cannot be anchored at both ends. But if it's missing some coils in the middle, it can still work reasonably well," says Krainer, who is testing this approach to treat a different disease, but was not involved in the research.

The treatment isn't a cure, but it may help people live longer with less disability.

"You're essentially converting a very severe patient to a Becker patient," says study researcher Ryszard Kole, PhD. Becker muscular dystrophy is a milder form of the disease.

Kole developed the theory behind the treatment while a professor of pharmacology at the University of North Carolina at Chapel Hill. He is now a senior vice president at AVI BioPharma, the company that's developing the treatment.

First Human Tests The study, which is published in The Lancet, tested increasing doses of the artificial nucleotides in 19 Duchenne patients, who ranged in age from 5 to 15.

The nucleotides were matched to the exact kind of genetic defect that was shared by all the patients, and they were given by weekly intravenous infusion.

Researchers tested samples of muscle tissue before and after the treatments to check dystrophin levels.

Seven patients, nearly all of them on the two highest doses of the drug given in the study, saw their dystrophin protein levels increase on the treatment.

Two of the best responders saw their levels climb from 0.9% to 17% and from 2% to 18%.

"There really is reason to be excited," says Richard Moxley III, MD, a pediatric neurologist at the University of Rochester. Moxley treats patients with muscular dystrophy and helped pioneer the use of corticosteroids in the disease. He was not involved in the current research.

http://www.webmd.com/parenting/news/20110725/gene-therapy-may-help-muscular-dystrophy-patients?src=RSS_PUBLIC

	Gene Therapy May Help Muscular Dystrophy Patients Tue Jul 26, 2011 2:54 am

Author

Message

Special Offers!

萬人迷

Joined: 06 Nov 2009

Posts: 2981

Post subject:

Medical Info Blog

蔡卓妍正妹露點自拍沈卓盈沙雪edk binhuorenti
樱井莉亚 free porn 河利秀日本av 白歆惠 filterui:imagesize-wallpaper ?剁??濠?瑁镐?
寮???涓榛?? inoue waka 無毛小穴熟妇孙艾丽 www,avrtys.com
青春美鲍美尻 luxuezipai 鬼妹娌宠タ??瀛? 蹇?? ??淇??╄阿姊
 亚洲原创无码区转贴春暖花开日本援交妹人体艺术#pn8 ed2k 黑崎扇菜
瑤瑤 tina yuzuki sex Erika Kimisita 公下エリカ女人的奶眼神
?︽??? 杞︽ā www.57vvv.com http.//www.95jq.com 姬川丽下载网盘 www.rtys8.cc
林私锺 45gdy 肉劉心悠斗轉星移合成照春暖花开SEX8亚洲无
風俗 ?у?ц???????? 56ALAV 學生自拍露點 Zouguang ctdisk
xvediox 255i 小松彩夏美女照 www.sex8.cc@gmail,com

Page 1 of 1

Theme designed by What Is Real!? 2005

All times are GMT + 8 Hours