As many as 40 rare diseases originate in repeating DNA sequences, these most often involving trinucleotide repeats. The repeats may be in coding or noncoding regions but they share the common feature that they are stable until a threshold length is reached, where progressive expansion occurs with disease symptoms. The disease mechanism in some cases arises from the expanded RNA transcript. This talk will focus on the rational design of small molecules to target these repeating sequences as well as a multivalent strategy to increase targeting efficiency. Strategies for using the multivalent approach while allowing cell permeability is a major theme and several novel approaches to develop cell permeable agents that function in vivo will be presented.

Rational design of cell-permeable, multivalent ligands for repeat disease RNA

255th National Meeting (2018)

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