Compositions and methods for modulating gene expression

C - Chemistry – Metallurgy – 07 – K

Patent

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C07K 7/64 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) C07H 21/00 (2006.01) C12N 15/63 (2006.01) C12Q 1/68 (2006.01) C07H 17/02 (2006.01)

Patent

CA 2649114

A description of novel artificial transcription factors (ATFs) is provided having a non- peptidic DNA-binding domain, flexible linker, and an effector domain based on a small molecule compound. These ATFs are capable of modulating transcription from nucleic acids both in vitro and in vivo. Importantly, these novel ATFs are capable of targeting and modulating the transcription of native (endogenous) genes in vivo. Method for targeted regulation of gene expression and the development of new class of pharmaceuticals are also provided.

La présente invention concerne des facteurs de transcription artificiels (Artificial Transcription Factors; ATF) innovants qui comprennent un domaine de liaison à l'ADN non peptidique, un lieur flexible et un domaine effecteur basé sur un composé de petites molécules. Ces ATF sont capables de moduler la transcription des acides nucléiques in vitro comme in vivo. Fait notable, ces ATF innovants sont capables de cibler in vivo des gènes natifs (endogènes) pour moduler leur transcription. L'invention concerne également un procédé de régulation ciblée de l'expression de gènes et le développement d'une nouvelle classe de produits pharmaceutiques.

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