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:6/26/2016)... Charlotte, NC (PRWEB) , ... June 26, 2016 , ... Brent Kasmer, a legally blind ... able to be personalized through a fitness app. The fitness app plans to fix the ... solutions currently only offer a one size fits all type program , They ...
(Date:6/25/2016)... ... June 25, 2016 , ... ... issues and applications at AcademyHealth’s Annual Research Meeting June 26-28, 2016, at the ... several important health care topics including advance care planning, healthcare costs and patient ...
(Date:6/25/2016)... ... ... closing of Bruton Memorial Library on June 21 due to a possible lice infestation, as ... of head lice: the parasite’s ability to live away from a human host, and to ... one in the event that lice have simply gotten out of control. , As lice ...
(Date:6/25/2016)... ... June 25, 2016 , ... First Choice Emergency Room ... Dr. Sesan Ogunleye, as the Medical Director of its new Mesquite-Samuell Farm facility. ... of our new Mesquite location,” said Dr. James M. Muzzarelli, Executive Medical Director of ...
(Date:6/25/2016)... ... June 25, 2016 , ... Dr. Calvin Johnson ... Recently, he has implemented orthobiologic procedures as a method for treating his patients. ... the first doctors to perform the treatment. Orthobiologics are substances that orthopaedic surgeons ...
Breaking Medicine News(10 mins):
(Date:6/23/2016)... Research and Markets has announced the addition of ... report to their offering. ... kidney failure, it replaces the function of kidneys by removing ... thus the treatment helps to keep the patient body,s electrolytes ... Increasing number of ESRD patients & substantial healthcare expenditure ...
(Date:6/23/2016)... 2016 Research and Markets has ... by Type (Organic Chemical (Sugar, Petrochemical, Glycerin), Inorganic Chemical), ... Parenteral) - Global Forecast to 2021" report to ... The global pharmaceutical excipients market is projected to reach ... 6.1% in the forecast period 2016 to 2021. ...
(Date:6/23/2016)... 23, 2016 Roche (SIX: RO, ROG; OTCQX: ... its Elecsys BRAHMS PCT (procalcitonin) assay as a dedicated ... shock. With this clearance, Roche is the first IVD ... solution for sepsis risk assessment and management. ... and PCT levels in blood can aid clinicians in ...
Breaking Medicine Technology: