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genetic therapy
- Question
- Hello,
I wanted to have news about research on gene therapy
- Answer
- Hello,
Currently a major player in this field of research, the UK CF Gene Therapy Consortium (www.cfgenetherapy.org.uk/) has developed a vector capable of carrying a healthy gene (producted in laboratory) to the lung cells of CF patients. This "carrier" is actually a particle of fat called a liposome that integrates into the cell membrane. This hopeful therapy has been tested in recent years by inhalation in humans and, except for some cases of fever, it was well tolerated. Regarding these encouraging results, the Consortium has planned to launch a major study in 2012 involving 120 English patients, adults and children over 12 years, with all types of mutations. Half of them will receive several doses of the gene by inhalation using a nebulizer called AeroEclipse and the other half a placebo. The administration will be once a month for a year and each therapy session will last approximately 40 minutes.
We have to wait until 2014 for results.
Best regards
Anna Ronayette - 25.06.2012
