Navigation Links
Molecular therapy for spinal muscular atrophy closer to clinical use
Date:12/16/2008

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
JacksonKN@missouri.edu
573-882-8353
University of Missouri-Columbia
Source:Eurekalert

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:
*Name:
*Comment:
*Email:
(Date:4/24/2017)... ... April 24, 2017 , ... ... opioid overdose deaths now claim the lives of 62 Americans each day.(1) Yet ... against drug manufacturers, distributors, pharmacies and prescribers by more than half.(2) Novus ...
(Date:4/24/2017)... ANGELES, CA (PRWEB) , ... April 24, 2017 , ... ... picture is worth a thousand words. The good news for single women is that ... for Attraction," which is available on April 25th. Joan's insight, personal experiences and sparkling ...
(Date:4/24/2017)... ... ... “Reflections of God’s Work”: an enlightening collection of life lessons leading each ... published author, Jerri Broglin, a survivor of great loss who gained insight on how ... for those searching for answers, as we are finding the answers that are so ...
(Date:4/24/2017)... NC and Seattle, WA (PRWEB) , ... April ... ... utilized by 75+ leading lifestyle brands, work wear distributors and International relief organizations ... they can carry. The company also offers lifestyle and work wear collections via ...
(Date:4/22/2017)... ... April 22, 2017 , ... Ecommerce sales have grown every year since ... $394.9 billion. The consequences of rapid innovation and growth are often neglected in ... it is every business and individual’s job to give something back to the planet ...
Breaking Medicine News(10 mins):
(Date:4/20/2017)... Inc ., a digital health company focused on modernizing and ... $25 million in Series B funding led by Qiming US ... Seattle that is part of a broader family ... included participation from SR One , who led ZappRx,s ... Google Ventures). As part of the financing, Mark McDade ...
(Date:4/20/2017)... Research and Markets has announced the ... Application Analysis, Regional Outlook, Growth Trends, Key Players, Competitive Strategies ... ... was valued at US$ 7,167.6 Mn in 2015, and is ... a CAGR of 5.6% from 2016 to 2024. ...
(Date:4/20/2017)... 20, 2017 NeuroVive Pharmaceutical AB (Nasdaq ... positive preclinical results demonstrating anti-fibrotic effects with ... steatohepatitis (NASH), in an additional well-validated experimental ... has previously shown similar anti-fibrotic effects in ... scientists present novel data demonstrating anti-fibrotic effects ...
Breaking Medicine Technology: