A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 31/713 (2006.01) A61K 31/7076 (2006.01)
Patent
CA 2602120
The present disclosure describes methods for altering the expression of a target gene comprising a rare cluster of codons, including, but not limited to, trinucleotide repeats. The method utilizes, in part, on amino acid deprivation or the limiting of specific charged tRNAs. The methods for altering target gene expression may be used in treatment methods to treat diseases in a subject organism in need of such treatment. Such methods for altering target gene expression have not been heretofore recognized in the art. Exemplary diseases that may be treated using the methods of the present disclosure include any disease where altering the expression of the target gene would provide treatment. Such diseases include all forms of cancer, ageing, infectious disease, metabolic disorders, inflammation, neurological disorders, diabetes, psychiatric disorders and diseases associated with trinucleotide repeats.
L'invention concerne des procédés permettant de modifier l'expression d'un gène cible comprenant un cluster rare de codons, entre autres, des répétitions trinucléotidiques. Ce procédé utilise, en partie, la privation d'acides aminés ou la limitation d'ARN de transfert chargés spécifiques. Ces procédés de modification de l'expression d'un gène cible peuvent être mis en oeuvre dans des méthodes de traitement visant à traiter des maladies dans l'organisme de patients nécessitant un tel traitement. Des exemples de maladies pouvant être traitées par la mise en oeuvre des procédés selon l'invention comprennent toutes les maladies susceptibles d'être traitées par la modification de l'expression d'un gène cible. Ces maladies comprennent toutes les formes de cancer, le vieillissement, les maladies infectieuses, les troubles métaboliques, les inflammations, les troubles neurologiques, le diabète, les troubles psychiatriques et les maladies associées à des répétitions trinucléotidiques.
Crouse Andrew B.
Detloff Peter J.
Fogler Rubinoff Llp
The Uab Research Foundation
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