Dr. Alton believes that this study, while modest in benefit, provides proof of concept that gene therapy should be further developed as a tool for treating CF. The next steps would involve pushing the boundaries to see if further benefit might be obtained with higher dosages. Also, if gene therapy is able to activate a small amount of CFTR in the lungs, there is the possibility that the chloride transport by this small amount of CFTR could be enhanced with the addition of a small molecule potentiator such as Kalydeco.
While gene therapy is an exciting concept, it is something that researchers have been working on for decades--basically since the discovery of the CF gene in 1989. The treatment benefits seen in the trial were modest, but represent the greatest success to date using this approach. Dr. Alton seems determined to continue down this road, but I have doubts... Personally, I think that the gene-editing or mRNA editing techniques (such as CRISPR-cas9 that we will hear about later in the conference) hold more promise as a way of actually changing an individual's genetic expression. Just my opinion. I was hoping for much better data after so many years of development.
Hormone Fluctuations Correlate with Respiratory Symptoms in Patients with Cystic Fibrosis. Presented by Raksha Jain.
Existing data has suggested that women with CF have worse health outcomes than men. Women demonstrate earlier colonization with respiratory pathogens and an increased exacerbation rate near ovulation. This study examined the role that sex hormones play in CF.
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