The current U.K. trial is again using fats, or lipids, but the DNA includes a segment called a promoter that should make gene expression last longer, says Eric Alton of Imperial College London, who heads the UK Cystic
Fibrosis Gene Therapy Consortium.
Despite expectations of a rapid breakthrough, no cystic
fibrosis gene therapy trial so far has been able to show long - term clinical improvement.
Not exact matches
When conventional wisdom said only
gene therapy could correct the root cause of cystic
fibrosis, Vertex developed pill - based treatments that address the underlying disease mechanism, says Altshuler.
Despite much effort, no one has managed to develop an effective form of
gene therapy to treat the lung problems of people with cystic
fibrosis.
Regulators in the US could soon be asked to approve a human trial of
gene therapy for cystic
fibrosis that uses a hybrid of the HIV and Ebola viruses.
The second team is lead by Bob Williamson at St Mary's Hospital Medical School and Duncan Geddes at the Royal Brompton Hospital, both in London, who want to test an experimental
gene therapy for cystic
fibrosis.
Building on these successes,
gene therapy may soon be used to correct hereditary genetic diseases like cystic
fibrosis, hemophilia, and Tay - Sachs and to activate the immune response against a wide variety of infectious diseases and cancers.
The method, reported in the November issue of Nature Biotechnology, could lead to safe and effective human
gene therapies for cystic
fibrosis, hemophilia, and a variety of other diseases.
«First trial of
gene therapy for cystic
fibrosis to show beneficial effect on lung function.»
A
therapy that replaces the faulty
gene responsible for cystic
fibrosis in patients» lungs has produced encouraging results in a major UK trial.
One early success story is cystic
fibrosis: since the
gene was identified in 1989, exceptional progress has been made in devising new experimental treatments, including
gene therapy trials.
There are now more than 100
gene therapy clinical trials aimed toward cancer, genetic diseases (such as ADA deficiency, cystic
fibrosis and hemophilia A), infectious diseases (including AIDS) and autoimmune diseases (such as rheumatoid arthritis).
Under the proposed law,
gene therapy will be approved only for the treatment of people with genetic diseases such as cystic
fibrosis and will not be allowed in germ cells, where genetic alterations would be passed on to the next generation.
WOMEN with cystic
fibrosis are to join British
gene therapy trials for the first time.
Patients undergoing
gene therapy for cystic
fibrosis at the Royal Brompton Hospital in London, for example, have not been confined to the laboratory.
Their findings, published last week in the Proceedings of the National Academy of Sciences, may provide clues for understanding how some forms of human lung cancer initiate and may also aid in the development of tools for successful
gene therapy of lung diseases such as cystic
fibrosis.
Cystic
fibrosis may not be the most likely candidate for
gene therapy, however, because the disease affects multiple organ systems.
His group began studying JSRV for it's potential applications for
gene therapy for lung diseases including cystic
fibrosis, provided the oncogenic potential of the virus can be controlled, Miller said.
A handful of biotechnology startups are vying to bring CRISPR - based
therapies to market, hoping to cure cystic
fibrosis and certain forms of blindness, among other conditions, by getting rid of the bad
genes that cause them.
«Now we are closer to understanding why patients with the exact same genetic mutation in the cystic
fibrosis gene have such widely varying manifestations of lung disease, and closer to finding new
therapies.»