Medical Science 25
Ion Channel Mutations: The Complexities of Treatment
July 24, 2024
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Arguably, the most challenging of targets for which we develop therapeutics are ion channels.
Mutations affecting ion channels are the most common cause of ‘applications for treatment’ submitted to n-Lorem. These channels regulate the passage of essential electrically charged ions, like sodium, potassium, calcium, and chloride, into cells. Alas, ion channels pose a significant challenge in the ASO discovery process, as there is little room for error due to the need for highly allele-selective ASOs to achieve success. Let’s ‘dive into the channel’ and explore the complexities of treating patients with ion channel mutations.
Recently a report on one of our patients with an ion channel mutation was published in Endpoints News highlighting the power of our technology for these disorders. Check it out by clicking the link below.
On This Episode We Discuss:
- Defining Ion Channels and Ions
- Health, Homeostasis, and Biological Buffering
- Multiple forms of the same gene
- Multiple Isoforms of Gene Product from the same gene
- Network redundancy
- Ion Channels are different
Watch on YouTube:
The host of the show is Dr. Stanley Crooke, a scientist, a physician, an entrepreneur and the father of antisense technology. Dr. Crooke is responsible for driving the development of antisense or ASO technology, an RNA-targeted technology responsible for the commercialization of three best- and first-in class medicines and more than 40 drugs in development. In 2020, Stan formed n-Lorem to use this powerful technology to develop experimental personalized ASO medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.
Credits:
Hosted by: Dr. Stan Crooke.
Videographer: Jon Magnuson of Mighty One Productions.
Producers: Colin Delaney, Kira Dineen, Jon Magnuson, Andrew Serrano and Amy Williford
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