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Application filed by Intellia Therapeutics IncfiledCriticalIntellia Therapeutics Inc
Publication of AR115505A1publicationCriticalpatent/AR115505A1/en
Micro-Organisms Or Cultivation Processes Thereof
(AREA)
Pharmaceuticals Containing Other Organic And Inorganic Compounds
(AREA)
Abstract
Esta divulgación se relaciona con guías modificadas de ARN que tienen actividad mejorada in vitro e in vivo en métodos de edición génica. Reivindicación 1: Un ARN guía (ARNg) que es un ARN guía simple corto (ARNsg corto) que comprende una porción conservada de un ARNsg que comprende una región horquilla, donde la región horquilla carece de al menos 5 - 10 nucleótidos y donde el ARNsg corto comprende una modificación en el extremo 5’ o una modificación en el extremo 3’.This disclosure relates to modified RNA guides that have improved in vitro and in vivo activity in gene editing methods. Claim 1: A guide RNA (gRNA) which is a short simple guide RNA (short sgRNA) comprising a conserved portion of a sgRNA comprising a hairpin region, where the hairpin region lacks at least 5-10 nucleotides and where the sgRNA short comprises a modification at the 5 'end or a modification at the 3' end.
ARP190101567A2018-06-082019-06-07
MODIFIED GUIDELINES FROM RNA FOR GENE EDITING
AR115505A1
(en)
mirna type artificial match, composition and method for suppressing expression of a target gene, pharmaceutical composition, method for treating a disease, and single-stranded nucleic acid