Oligonucleotide binding agents

A method for validating a candidate lead compound and identifying the site of candidate lead compound-microRNA interaction comprising:

• identifying binding of a candidate lead compound to a microRNA or a microRNA fragment;
• altering the sequence of the microRNA or microRNA fragment via introduction of one or more point mutations, thereby generating a mutated microRNA or a mutated microRNA fragment;
• and
• identifying absence of binding of the candidate lead compound to the mutated microRNA or mutated microRNA fragment according to the method of any of the preceding claims,
• thereby validating the candidate lead compound and identifying the site of candidate lead compound-microRNA interaction.

A method for validating a candidate lead compound and identifying the site of activity of the candidate lead compound within the microRNA pathway comprising:

• identifying binding of a candidate lead compound to a microRNA or a microRNA fragment according to any of the methods of the preceding claims;
• assaying levels of pri-microRNA, pre-microRNA and mature microRNA in the presence of the candidate lead compound, as compared to in the absence of the candidate lead compound;
• and
• identifying the site of activity of the candidate lead compound within the microRNA pathway based upon the relative levels of pri-microRNA, pre-microRNA and mature microRNA assayed in the presence of the candidate lead compound, as compared to in the absence of the candidate lead compound,
• thereby validating the candidate lead compound and identifying the site of activity of the candidate lead compound within the microRNA pathway.