Navigation Links
Molecular therapy for spinal muscular atrophy closer to clinical use

COLUMBIA, Mo. Spinal muscular atrophy, a neurodegenerative disorder that causes the weakening of muscles, is the leading cause of infant death and occurs in 1 in 6,000 live births. While trans-splicing (a form of molecular therapy) has had impressive results as a treatment for spinal muscular atrophy in cell-based models of disease, scientists have been unable to translate the therapy to the human body. A University of Missouri researcher has developed a strategy that will enhance trans-splicing activity and bring it closer to being used in the clinical setting.

Spinal muscular atrophy is caused by the loss of survival motor neuron-1(SMN1). In humans, a nearly identical copy gene is present called SMN2. Because of a single molecular difference, SMN2 alone cannot compensate for the loss of SMN1, but it can be used as a primary target for therapeutics, including trans-splicing. Trans-splicing therapy relies on splicing, or uniting, of mutant RNA and therapeutic RNA in order to correct RNA sequence.

To improve efficiency, the researchers developed a trans-splicing system that uses a strand of RNA that can bind to a gene and inactivate it. Turning the gene "off" reduces competition at splice sites and improves the likelihood of achieving the desired results.

"The key to introducing trans-splicing in clinical settings is developing efficient trans-splicing systems," said Chris Lorson, investigator in the Christopher S. Bond Life Sciences Center; associate professor of veterinary pathobiology in the MU College Veterinary Medicine; and scientific director for Fight SMA, a private spinal muscular atrophy research foundation in Richmond, Va. "We have found that reducing the competition between the splice sites enhances the efficiency of trans-splicing. This strategy provides insight into the trans-splicing mechanism and significantly improves trans-splicing activity in a mouse model of spinal muscular atrophy. "


Contact: Kelsey Jackson
University of Missouri-Columbia

Page: 1

Related medicine news :

1. A new molecular zip code, and a new drug target for Huntingtons disease
2. New knock-out gene model provides molecular clues to breast cancer
3. Molecular probe paints cancer cells in living animals, Stanford researchers find
4. Story ideas from molecular & cellular proteomics
5. SNM seeks novel approaches to molecular imaging to showcase at annual meeting
6. SNM seeks novel approaches to molecular imaging to showcase at annual meeting
7. Molecular fingerprint of breast-cancer drug resistance can predict response to treatment
8. Molecular profiling can accurately predict survival in colon cancer patients
9. Xceed Molecular Names Fran Tuttle to Its Board of Trustees
10. Virginia G. Piper Charitable Trust and Flinn Foundation launch molecular diagnostics initiative
11. Xceed Molecular Launches Ziplex(R) Automated Gene-Expression System at the Association for Molecular Pathology, Booth 84/85
Post Your Comments:
(Date:11/30/2015)... (PRWEB) , ... November 30, 2015 , ... ... Amanda Beard and Brooke Bennett are collaborating with brands across various categories through ... four influential figures make up an elite group of Gold Medal Moms who ...
(Date:11/30/2015)... Turks & Caicos Islands, BWI (PRWEB) , ... December 01, 2015 ... ... in 2015. Recently Caribbean Journal, one of the leading digital news sites highlighting ... The top reason? The weather. While much of North America shivers under chilly ...
(Date:11/30/2015)... Garden City, NY (PRWEB) , ... December 01, 2015 , ... ... as a 2015-2016 inductee into its VIP Woman of the Year Circle. She ... is the nation’s leading networking organization exclusively for professional women, boasting more than 850,000 ...
(Date:11/30/2015)... ... 30, 2015 , ... 1Heart Caregiver Services’ Executive Team actively ... Philippine American Chambers of Commerce held from November 6-8, 2015 at the Monte ... as an active delegate from the Philippine American Chamber of Commerce of Greater ...
(Date:11/30/2015)... , ... November 30, 2015 , ... ... also extremely stressful. At the VA Maryland Health Care System, the Caregiver ... veterans patients. “Caregivers have a difficult job. Seventy-four percent report that their ...
Breaking Medicine News(10 mins):
(Date:12/1/2015)... , Dec. 01, 2015 ... announced the addition of the ... Campylobacter, Cryptosporidium, E. Coli, Enterovirus, Rhinovirus, ... report to their offering. --> ... addition of the "2016 Europe ...
(Date:12/1/2015)... December 1, 2015 ... Topical Contraceptives, Male Condoms, Female Condoms, Intrauterine ... Contraceptive Diaphragms, Contraceptive Sponges, Non-Surgical Permanent Contraception ... Growth, Trends and Forecast 2014 - 2020 ... Research (TMR).The report states that the global ...
(Date:12/1/2015)... Pa. , Dec. 1, 2015  Today, ... announced the launch of CareFront, a first-of-its-kind population ... patients diagnosed with cancer. Designed to be built ... diagnosed cancer patients with resources for their care ... The program also offers tools to help patients ...
Breaking Medicine Technology: