Methods for the identification and validation of functional intracellular targets with intramers or in vivo selection

摘要

Described are methods for the identification and validation of (1) functional intracellular targets and (2) nucleic acids (intramers) which are capable of binding to and modifying the function of said intracellular targets. These nucleic acids or, alternatively, compounds showing the same or a similar target specificity and/or effect are useful for the preparation of therapeutics, particularly for gene therapy.