Navigation Links
Save messengers -- modified mRNAs open up new therapeutic possibilities
Date:2/7/2011

Defects in the genome are the cause of many diseases. Gene therapy direct replacement of mutant genes by intact DNA copies offers a means of correcting such defects. Now a research team based at the Medical Center of the University of Munich, and led by Privatdozent Dr. Carsten Rudolph, has taken a new approach that avoids DNA delivery. The team shows for the first time that chemical modification of mRNAs (the metabolically active molecules derived from genomic DNA that programs protein synthesis) provides a promising alternative to DNA-based procedures. In contrast to the latter, the modified RNAs do not increase the risk of cancer, and do not induce frequently observed severe immune reactions seen with DNA or unmodified mRNA. The researchers show that the new method is life-saving in mice with a congenital lethal lung defect. "These results clearly demonstrate the therapeutic potential of our mRNAs," says Rudolph. (Nature Biotechnology, 7 February 2011)

Gene therapy offers great potential for the treatment of both congenital and acquired diseases that are otherwise not amenable to other treatments. However, the approaches that have been tested so far are associated with serious side-effects. The use of engineered viruses to introduce intact genes into the cellular genome are associated with an increased risk of developing leukaemia, and can provoke strong immune reactions. Nonviral vectors, on the other hand, tend to be inefficient delivery vehicles. The new RNA-based method displays neither of these disadvantages. "Chemical modification of the mRNA prevents it from activating the immune system, so that no inflammatory reaction ensues," says Rudolph. "Furthermore, in contrast to conventional mRNA, the modified mRNA can be administered repeatedly, is more stable and is effective at very low doses."

The new method, referred to as Transcript Therapy by its inventors, opens up new opportunities for targeted treatments. The modified mRNAs shuttle genetic information into cells in a form that can be used directly for the production of therapeutically active proteins. In addition to their potential for the treatment of genetic diseases, they represent an alternative to established protein-based treatment regimes. The researchers demonstrated the efficacy of the latter application in a mouse model. A single intramuscular injection of mRNAs coding for the hormone erythropoetin, which stimulates the differentiation of red blood cells, led to a significant increase in erythrocyte counts four weeks later.

The team also employed a mouse model to show that the technique can effectively be used to treat a genetic disease. For this, they chose a strain with a lethal congenital lung defect, caused by its inability to make surfactant protein B (SP-B). Regular application of mRNA for SP-B, as an aerosol, to the lungs of the mutant mice restored lung function, and the effect persisted for the duration of the study. Rudolph sees great scope for transcript therapy in regenerative medicine and the treatment of metabolic diseases, and hopes to test the efficacy of modified mRNAs in a clinical setting over the next few years.


'/>"/>

Contact: Dr. Carsten Rudolph
Carsten.Rudolph@med.uni-muenchen.de
49-895-160-7711
Ludwig-Maximilians-Universitt Mnchen
Source:Eurekalert

Related medicine news :

1. NIH Stem Cell Guidelines Should Be Modified, UCSF Team Reports
2. Adults demonstrate modified immune response after receiving massage, Cedars-Sinai researchers show
3. New On the Menu: Genetically Modified Salmon?
4. FDA Advisers Weigh Approval of Genetically Modified Salmon
5. FDA Advisers Consider Approval of Genetically Modified Salmon
6. Consumer Activists Want Modified Salmon to Be Labeled
7. New therapeutic target for most common solid cancer in childhood?
8. UC studies show marijuana has therapeutic value, reports to legislature
9. United Therapeutics Corporation Withdraws European Marketing Authorization Application for Tyvaso for the Treatment of Pulmonary Arterial Hypertension
10. Elements Therapeutic Massage Announces Franchise Expansion Plan for Massachusetts
11. Castrate-Metastatic Prostate Cancer: Therapeutic Considerations for Advanced Disease
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/9/2016)... ... February 09, 2016 , ... ... businesses recover after a disaster, announced today the acquisition of Hawaii DKI. Hawaii ... , “Investing in like-minded companies who excel at service and response ...
(Date:2/9/2016)... ... February 09, 2016 , ... Cirracore Enterprise ... enterprises move workloads to the cloud. Cirracore provides a secure VMware® vCloud ... traversing the Internet. Transformation Solutions (TSL Partners) provides a full range of ...
(Date:2/9/2016)... VA (PRWEB) , ... February 09, 2016 , ... On ... The family scheduled an appointment after they noticed their furnace not producing any heat. ... cracked heat exchanger. The cracked heat exchanger was leaking dangerous levels of carbon monoxide ...
(Date:2/9/2016)... ... February 09, 2016 , ... ZipHearing.com ... access to affordable hearing aids , increase industry transparency, and promote awareness ... United States. , “For the average consumer, the hearing aid industry is esoteric ...
(Date:2/9/2016)... ... February 09, 2016 , ... United Methodist Communications collaborated with ... an animated video designed to prevent the next widespread Ebola outbreak ... are being distributed throughout Togo, Liberia, Sierra Leone, Nigeria, Côte d’Ivoire and other ...
Breaking Medicine News(10 mins):
(Date:2/9/2016)... , Feb. 9, 2016  Johnson & Johnson (NYSE: ... Markets Global Healthcare Conference on Tuesday, Feb. 23, at ... Dominic Caruso , Vice President, Finance & Chief Financial ... will represent the Company in a session scheduled at ... --> www.investor.jnj.com . --> This ...
(Date:2/9/2016)...  The Parenteral Drug Association, Inc. (PDA), the ... sterile drug products, today announced the publication of ... With link to Comparison Spreadsheet. The document ... FDA, the EU, the Pharmaceutical Inspection Convention/Scheme and ... --> PDA,s Global Sterile Task ...
(Date:2/8/2016)... 2016 Aesthetic Devices - Medical Devices Pipeline ... report, "Aesthetic Devices - Medical Devices Pipeline Assessment, 2015" ... stage. This report is prepared using ... by GlobalData,s team of industry experts. *Note: Certain ... based on the availability and relevance of data in ...
Breaking Medicine Technology: