Guest guest Posted January 22, 2003 Report Share Posted January 22, 2003 Splicing technique treatment hope for disease BYLINE: Health Newswire reporters HIGHLIGHT: US scientists believe that they are making progress in developing treatments for genetic diseases such as breast cancer, muscular dystrophy and cystic fibrosis. Their theory is centred on a defect in a complex process known as " pre-messenger RNA splicing " which takes the information coded in genes and makes it available for building proteins. Dr Krainer, a molecular biologist at New York's Cold Spring Harbor Laboratory, and colleagues explain that they have found a way to correct RNA splicing defects that are implicated in diseases such as breast cancer and the neurodegenerative disease spinal muscular atrophy. Writing in the journal Nature Structural Biology, they say that their technique could make it possible to correct RNA splicing defects associated with any gene or disease. They hope that their research can potentially solve the problem of improperly spliced mature messenger RNA (mRNA), which causes disease by rendering proteins incapable of performing their job properly. Dr Brenton Graveley, of the University of Connecticut Health Center, who is familiar with the research but not involved with it, said that the new method of correcting RNA splicing deficits is " very promising " . He said, " There are a lot of hurdles to be overcome in terms of delivering the corrective molecules to the cells that need to be treated. But theoretically the exact same approach could be taken for any gene at all, and the list of genes that have defects at the level of RNA splicing is very long. " Becki YOUR FAVORITE LilGooberGirl YOUNGLUNG EMAIL SUPPORT LIST www.topica.com/lists/younglung Pediatric Interstitial Lung Disease Society http://groups.yahoo.com/group/InterstitialLung_Kids/ Quote Link to comment Share on other sites More sharing options...
Guest guest Posted February 8, 2003 Report Share Posted February 8, 2003 Here is a copy of an article that Becki sent on this issue back in October. --------- Forwarded message ---------- From: MissGooberGirl@... To: younglung@..., younglungonlinecommunity@..., lung-parents , cfparents , cysticfibrosis@... Date: Tue, 22 Oct 2002 11:41:37 EDT Subject: Gene Therapy Article Message-ID: Sunday, 20 October, 2002, 23:04 GMT 00:04 UK Gene therapy targets cystic fibrosis Cystic Fibrosis is caused by a single defective gene Scientists believe they may be able to overcome a natural body defence which is hindering gene therapy for cystic fibrosis. Thousands in the UK are battling the lung disease, which, without transplantation, can prove fatal before the age of 30. Gene therapy is one of the big hopes for future treatments, as a mutation in just one gene causes a malfunction within cells on the lung surface. The malfunction leads to overproduction of mucus, which clogs the lung and makes sufferers vulnerable to chronic infections. Because the surface of the lung is relatively accessible compared with other internal organs, it is hoped that a gene therapy could be inserted into an inhaler to replace the mutated gene. However, trials of gene therapy have only had limited success so far, with the effects often proving short-lived. This is partly due to the natural defences of the lungs, which are primed to defend their surface against all sorts of allergens, dust particles, viruses and other foreign particles. This defence system proves pretty effective against the viruses used to deliver the new gene into cells. Keeping therapy working Now a team of researchers from the Women's and Children's Hospital in Adelaide, Australia, claim they have found a way of prolonging the effect of the treatment. As well as the therapy, they gave a dose of a detergent-like substance found naturally in the healthy lung. They believe this helps " condition " the surface of the lung and hold off the defence mechanism. So far, they have only tested their theory in the nasal passages of mice - which react in a similar way to the surfaces of the lungs. In their experiments, cells lining these airways not only responded to the gene therapy, but maintained that response - so far for 110 days. As nasal cells are generally replaced once every three months, this means it is possible that the change is appearing in newly-created cells as well as existing ones. Helpful HIV The team used a modified form of HIV to carry the therapy - it has been rendered incapable of causing an HIV infection, but can still enter cells and replace the key gene with a new, healthy sequence. Dr Parsons, who led the research, said: " Airway cells are replaced every three months so our findings are particularly exciting because they imply we are in fact targeting airway stem cells through this approach. " Some of the therapeutic gene must have been passed on from these parent stem cells to their daughter cells for the effect to persist beyond three months. " The next challenge is to take these successes and reproduce them in the lungs of real cystic fibrosis patients, and the Australian team is fine-tuning the technique so that human clinical trials can be started. Becki YOUR FAVORITE LilGooberGirl YOUNGLUNG EMAIL SUPPORT LIST www.topica.com/lists/younglung Pediatric Interstitial Lung Disease Society http://groups.yahoo.com/group/InterstitialLung_Kids/ Quote Link to comment Share on other sites More sharing options...
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