Navigation Links
Gene therapy for muscular dystrophy fixes frail muscle cells in animal model, Stanford study finds

A new gene therapy technique that has shown promise in skin disease and hemophilia might one day be useful for treating muscular dystrophy, according to a new study by researchers at Stanford University School of Medicine.

In the study, scheduled to be published online in the Proceedings of the National Academy of Sciences the week of Jan. 2, the researchers used gene therapy to introduce a healthy copy of the gene dystrophin into mice with a condition that mimics muscular dystrophy. The dystrophin gene is mutated and as a result produces a defective protein in the roughly 20,000 people in the United States with the most common form of the disease.

Using gene therapy to treat muscular dystrophy isn't a new idea. Thomas Rando, MD, PhD, associate professor of neurology and neurological sciences, said that researchers have tried several different techniques with variable success. One hurdle is getting genes into muscle cells all over the body. Another is convincing those cells to permanently produce the therapeutic protein made by those genes.

The gene therapy technique Rando and postdoctoral fellow Carmen Bertoni, PhD, used was developed by Michele Calos, PhD, associate professor of genetics. One of the main advantages of this method is that it could potentially provide a long-term fix for a variety of genetic diseases, including muscular dystrophy.

In muscular dystrophy, the muscle cells break down and are slowly replaced by fat. Eventually people with the disease are confined to a wheelchair and usually die in their 20s. There is currently no effective treatment for the disease, which explains why gene therapy remains a hope despite the significant hurdles.

Rando said the PNAS paper highlights an additional requirement for any gene therapy to be successful: the introduced gene must produce healthy dystrophin protein in large quantities in order to repair the entire muscle cell. Previous muscular dystrophy gene therapy studies
'"/>

Source:Stanford University Medical Center


Page: 1 2 3

Related biology news :

1. Adding Radiation Therapy To Chemotherapy Improves Survival In Patients With High-risk Breast Cancer
2. Columbia research lifts major hurdle to gene therapy for cancer
3. Combination therapy boosts effectiveness of telomere-directed cancer cell death
4. Gene therapy converts dead bone graft to new, living tissue
5. Study identifies predictors of HIV drug resistance in patients beginning triple therapy
6. New imaging method gives early indication if brain cancer therapy is effective, U-M study shows
7. Muscle-targeted gene therapy reverses rare muscular dystrophy in mice
8. New therapy for HIV/AIDS eliminates needles and excessive toxicity
9. New Treatment Rivals Chemotherapy For Lymphoma, Study Finds
10. Gene therapy for Parkinsons disease moves forward in animals
11. Pulsating ultrasound enhances gene therapy for tumors
Post Your Comments:
(Date:12/19/2014)...   LaunchKey , the first decentralized mobile authentication ... of Things era, today announced the close of $3 ... led by Metamorphic Ventures with participation from ENIAC Ventures, ... others.  LaunchKey has raised $4 million to date, and ... and bring LaunchKey to market in 2015. ...
(Date:12/17/2014)... Valencell, a leader in performance biometric data ... industry leaders such as Intel, Jabra, Atlas, and Scosche ... products. These products will be showcased at CES 2015, ... . "Our partners choose PerformTek ... – with the clinical data to back it up," ...
(Date:12/11/2014)... --Research and Markets , ... ) has announced the addition of the ... to their offering. One major ... technology. With continuous advances in technology, it is ... standard that meets the needs and expectations of ...
Breaking Biology News(10 mins):LaunchKey Raises $3 Million in Additional Funding Led by Metamorphic Ventures 2LaunchKey Raises $3 Million in Additional Funding Led by Metamorphic Ventures 3Valencell PerformTek Biometrics Power the Most Accurate Wearables at CES 2015 2Biometrics Market in the APAC Region 2015-2019: Key Vendors are 3M Cogent, NEC, Safran and Suprema 2
... release is available in French . ... action to better control the sale of chemicals that ... Lunn, Minister of Natural Resources, and the Honourable Stockwell ... regulations for these chemicals, known as explosives precursors, will ...
... American,Association of Airport Executives President Chip ... Traveler (RT) program with the opening of,RT ... Reagan,Washington National Airport., "The expansion of ... in,the nation,s capital will further highlight the ...
... — Robots are fluent in their native language of 1 ... imprecise nature of human language. While scientists are making slow, ... responds to speech, gestures and body language, a more straightforward ... homes sooner. A team of researchers led by Charlie ...
Cached Biology News:New regulations tighten controls on restricted chemicals 2AAAE Applauds Expansion of Registered Traveler Program to Nation's Capital 2AAAE Applauds Expansion of Registered Traveler Program to Nation's Capital 3Robot fetches objects with just a point and a click 2Robot fetches objects with just a point and a click 3Robot fetches objects with just a point and a click 4
(Date:12/22/2014)... 22, 2014 The American ... of original research, reviews and editorials addressing developments ... practice, today published a provocative article exploring the ... progression and potential treatment of prostate cancer. ... and proposes the possibility that there could be ...
(Date:12/22/2014)... 2014 Fertility Associates of Memphis has ... USA , a network of more than 20 contributing ... patients have fast access to a national pool of ... . Fertility Associates of Memphis is the only fertility ... option. , Dr. William Kutteh , director of ...
(Date:12/19/2014)... Charm Sciences is pleased to announce that ... Aflatoxin M1 in raw commingled milk is the first ... The peer reviewed report of the validation by Technology ... and Fisheries Research (ILVO-T&V) has been published by the ... most toxic aflatoxin and a known carcinogen, can be ...
(Date:12/19/2014)... 19, 2014 Naurex Inc., a biopharmaceutical company ... diseases of the central nervous system, today announced that ... officer, will present at the 33 rd annual ... place at 3:00 p.m. PST on Tuesday, January 13, ... San Francisco, Calif. About ...
Breaking Biology Technology:Prostate Cancer Experts Dissect the Role of Follicle-stimulating Hormone in the Development, Progression and Potential Treatment of Prostate Cancer 2Prostate Cancer Experts Dissect the Role of Follicle-stimulating Hormone in the Development, Progression and Potential Treatment of Prostate Cancer 3Prostate Cancer Experts Dissect the Role of Follicle-stimulating Hormone in the Development, Progression and Potential Treatment of Prostate Cancer 4Fertility Associates of Memphis Announces Partnership with Donor Egg Bank USA 2Fertility Associates of Memphis Announces Partnership with Donor Egg Bank USA 3Charm Sciences Achieves Independent Validation of First 15 Minute Quantitative Screening Method for Aflatoxin M1 2Naurex to Present at 33rd Annual J.P. Morgan Healthcare Conference 2
... , , BETHESDA, Md., July ... and NWBS) today announces that it will seek shareholder approval for ... AIM market ("AIM") (the "De-listing"). The Company plans to continue its ... Board Market ("U.S. listing"), and plans to file an S1 registration ...
... , SAN DIEGO, July 23 Anadys Pharmaceuticals, Inc. ... quarter 2009 financial results on Thursday, July 30, 2009, after the U.S. ... hold a conference call and webcast on Thursday, July 30, 2009 at ... results and highlights and to provide an update on its development programs. ...
... , SUNNYVALE, Calif., July 23 ... for the treatment of chronic total occlusions (CTOs), today announced the ... Chris previously held the position of Executive VP, Sales and Marketing ... Devices including the last 2 years at FlowCardia. , ...
Cached Biology Technology:Northwest Biotherapeutics Announces Plans to De-list From AIM and Consolidate its Stock Market Listing in U.S. 2Northwest Biotherapeutics Announces Plans to De-list From AIM and Consolidate its Stock Market Listing in U.S. 3Anadys Pharmaceuticals to Report Second Quarter 2009 Financial Results 2FlowCardia, Inc. Appoints Chris Barys to Chief Operating Officer 2
...
Request Info...
OmniSlide thermal Cycler for slide applications. 20 slide capacity. Humidity Chamber. Slide Rack can be transferred directly to Wash Module to avoid handling individual slides....
... effect inhibition assays, also known as CPE and ... supernatents; serum from PK studies and delivery formulations. ... as the quantity of interferon required per milliliter ... by 50%. All standards used in ...
Biology Products: