How Does It Work? | Individualized Oligonucleotide Therapies

In this week’s episode of our “How Does It Work?” video series, we explore Individualized Oligonucleotide Therapies.

Around 6 billion letters form the genetic code that makes each person’s biology unique. Genomic sequencing allows us to read this code to find defects that cause disease, enabling us to tailor therapies for individual patients.

This was first realized in 2019 for a 6-year old patient suffering from a life-limiting neurodegenerative disease. The patient’s genome sequence revealed that the mRNA from a critical gene was incorrectly spliced, which led to a defective protein inside of cell lysosomes. But a custom oligonucleotide was designed for the patient to mask the defective sequence, enabling normal splicing of the mRNA, thus a normal functioning protein to be formed.

Thousands of other rare diseases are also caused by gene mutations, including other genetic diseases and cancers. Oligonucleotides therefore hold enormous potential in this field, and we’re likely to see many more emerge for individual patients as this research continues.
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