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1. WO2016089866 - RNA-GUIDED SYSTEMS FOR IN VIVO GENE EDITING

Publication Number WO/2016/089866
Publication Date 09.06.2016
International Application No. PCT/US2015/063181
International Filing Date 01.12.2015
IPC
C12N 15/00 2006.1
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
02Preparation of hybrid cells by fusion of two or more cells, e.g. protoplast fusion
07Human cells
C12N 15/87 2006.1
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
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9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
10Transferases (2.)
A61K 48/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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A61K 48/005
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61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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102Mutagenizing nucleic acids
C12N 15/11
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
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63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
902using homologous recombination
907in mammalian cells
C12N 2310/20
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2310Structure or type of the nucleic acid
10Type of nucleic acid
20involving clustered regularly interspaced short palindromic repeats [CRISPRs]
Applicants
  • PRESIDENT AND FELLOWS OF HARVARD COLLEGE [US]/[US]
Inventors
  • WAGERS, Amy J.
  • TABEBORDBAR, Mohammadsharif
  • CHEW, Wei Leong
  • CHURCH, George M.
Agents
  • IWANICKI, John P.
Priority Data
62/085,78501.12.2014US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) RNA-GUIDED SYSTEMS FOR IN VIVO GENE EDITING
(FR) SYSTÈMES GUIDÉS PAR ARN POUR L'ÉDITION DE GÈNE IN VIVO
Abstract
(EN) Methods of editing target nucleic acids are provided using a guide RNA and a Cas9 protein to excise exons in a target gene and where the edited gene is expressed to produce a truncated polypeptide.
(FR) L'invention concerne des méthodes d'édition d'acides nucléiques cibles à l'aide d'un ARN guide et d'une protéine Cas9 pour exciser des exons dans un gène cible, le gène édité étant exprimé pour produire un polypeptide tronqué.
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