Here is a great article about getting closer to finding a CURE for CF!! YEA!!!! Which reminds me, we finally have a date for our Great Strides Walk that we are starting here in our town! It will be on Saturday June 13th! Unfortunately we will not be able to take any on-line donations this year since this is the first year for the Great Strides Walk in this area, so we are not in the system yet. But next year we will be;) On Monday 2/23 at 6:30pm, we are having a CF mom meeting with Erica, the Great Strides Coordinator, to go over more details. I'll update you later.
Cystic fibrosis gene cure closer:
Mucus in the lungs can lead to infection
US researchers say they are a step closer to developing a gene therapy cure for cystic fibrosis (CF).
In lab tests their altered virus effectively delivered a new gene to replace the faulty one that causes CF and completely rid the lung of disease.
Previous attempts have been hampered by the immune system mopping up the virus before it delivered its cargo.
The University of California work is published in Proceedings of the National Academy of Sciences.
Although the gene therapy is still in its infancy, it has already been used with some success in diseases such as haemophilia.
UK researchers plan to begin the first large scale trial of gene therapy in CF patients this year.
Proof of concept:
Around 8,000 people in the UK have CF and one in 25 are carriers for the condition. CF produces thick, sticky mucus that clogs the lungs and the pancreas, leading to life-threatening chest infections and problems with digestion.
Existing treatments only ease symptoms and life expectancy is low - most with CF die before they reach 40.
In the future it is hoped that gene therapy will provide a cure by replacing the defective CF gene which disables or destroys a protein known as CFTR.
In order to get the new, healthy gene into the cells, scientists use a harmless virus.
The University of California team, working with experts from the University of Iowa, found a way to make this virus more effective at entering lung cells.
More work is needed before this could be translated into clinical benefit for people with CF
A spokeswoman from the UK Cystic Fibrosis Gene Therapy Consortium
They mutated the adeno-associated virus to develop two major possessions - the ability to bind to different receptors or bind to a more plentiful receptor on the surface of lung cells and to make it past the cell surface membrane and into the cell.
Lead researcher Professor David Schaffer said: "In human lung tissue, it completely rescued the chloride ion transport properties of the cells after delivering the correct copy of the CFTR gene to replace the mutated copy of the gene that is present in CF patients."
They now plan to test the treatment further and to adapt the virus to gene therapy for other diseases, including Alzheimer's.
A spokeswoman from the UK Cystic Fibrosis Gene Therapy Consortium, which is trialling gene therapy in patients, said: "The techniques used in this research are innovative, but more work is needed before this could be translated into clinical benefit for people with CF.
"Although the results are positive in human lung tissue in a laboratory, it remains to be seen if it would work in the patient."
The UK CF Gene Therapy Consortium, funded by the Cystic Fibrosis Trust, are using a non-viral method to get the gene therapy product into the body and anticipate going to a major multi-dose clinical trial in 2010.After reading this article, how could you not donate to help the scientist continue to find the CURE for our Lil' Chris and for all CFers?!?!?!
If you would like to donate on Lil' Chris' behalf or join our walk, please
email me here.
Just remember...EVERY PENNY COUNTS!! I know all too well how hard times are right now, but we can't let that stop us. If everybody did, that could mean years off of Lil' Chris' life and all CFers lives.
Thank you in advance from the bottom of my heart,
M
PS. My MIL's Great Strides Walk will be on Saturday May 2nd. Please
email me if you are around the Ocean City Boardwalk and would like to attend.
PSS.
Click here if you still need convincing. It is an article of a 23 year old dying CFers wish for other CFers.
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