Methods for the identification and validation of functional...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/10 (2006.01) C12Q 1/68 (2006.01)

Patent

CA 2365297

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.

La présente invention concerne des méthodes d'identification et de validation (1) de cibles intracellulaires fonctionnelles et (2) d'acides nucléiques capables de se lier auxdites cibles intracellulaires et de modifier leur fonction. Ces acides nucléiques ou, alternativement, des composés présentant la même spécificité ciblée et/ou le même effet ciblé ou une spécificité ciblée similaire et/ou un effet ciblé similaire sont utilisés pour la préparation d'agents thérapeutiques, notamment pour la thérapie génique.

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