When results start coming through, there will be a belated realization of the potential.
There are currently around 7,000 known monogenic diseases caused by mutations in a single gene. However, a recent study identified 72 monogenic disorders that have a prevalence of at least 1 in 20,000 individuals in the human population. Of these, 34 are caused exclusively by mutations in a single gene. The number of known monogenic diseases continues to grow as gene discovery efforts advance, aided by technologies like whole exome/genome sequencing.
https://pubmed.ncbi.nlm.nih.gov/34515378/
Stoke therapeutics have identified approximately 1,200 monogenic diseases and approximately 6,500 additional genes with RNA target signatures which we believe to be amenable to TANGO.
When results start coming through, there will be a belated...
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