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1. WO2017205423 - PULMONARY TARGETED CAS9/CRISPR FOR IN VIVO EDITING OF DISEASE GENES

Publication Number WO/2017/205423
Publication Date 30.11.2017
International Application No. PCT/US2017/034070
International Filing Date 23.05.2017
IPC
C12N 15/861 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
861Adenoviral vectors
C12N 15/66 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
66General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
A61K 48/00 2006.01
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
C12N 15/10 2006.01
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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
10Processes for the isolation, preparation or purification of DNA or RNA
C12N 15/63 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
CPC
A61K 48/00
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
A61K 48/0008
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
0008characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
A61K 48/0058
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
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
A61K 48/0066
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
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
A61P 7/04
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
7Drugs for disorders of the blood or the extracellular fluid
04Antihaemorrhagics; Procoagulants; Haemostatatic agents; Antifibrinolytic agents
C07K 14/755
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
745Blood coagulation or fibrinolysis factors
755Factors VIII ; , e.g. factor VIII C (AHF), factor VIII Ag (VWF)
Applicants
  • WASHINGTON UNIVERSITY [US]/[US]
Inventors
  • CURIEL, David
Agents
  • ZACKSON, Saul
Priority Data
62/340,50123.05.2016US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) PULMONARY TARGETED CAS9/CRISPR FOR IN VIVO EDITING OF DISEASE GENES
(FR) CAS9/CRISPR DE CIBLAGE PULMONAIRE POUR L'ÉDITION IN VIVO DE GÈNES DE MALADIE
Abstract
(EN)
An adenovirus vector comprising: a pulmonary targeting coding sequence, CRISPR components such as a Cas9 coding sequence, and a guideRNA coding sequence which can be used for gene therapy. The adenovirus can be a gorilla adenovirus, and can include a pulmonary cell targeting sequence such as an MBP targeting ligand coding sequence. The adenovirus can be targeted to pulmonary epithelium with a vascular specific promoter and integrin targeting peptides incorporated into a viral knob. The adenovirus can be used to introduce serum proteins via the pulmonary epithelium. Hemophilia can be treated by adenoviral introduction of factor VIII or factor IX.
(FR)
L'invention concerne un vecteur d'adénovirus comprenant : une séquence de codage de ciblage pulmonaire, des constituants de CRISPR, tels qu'une séquence de codage de Cas9, et une séquence de codage d'ARN guide qui peut être utilisée pour une thérapie génique. L'adénovirus peut être un adénovirus de gorille et peut comprendre une séquence de ciblage de cellule pulmonaire telle qu'une séquence de codage de ligand de ciblage de PLM (peptide de liaison myéloïde). L'adénovirus peut être ciblé sur l'épithélium pulmonaire avec un promoteur spécifique vasculaire et des peptides ciblant l'intégrine, incorporés dans un bouton viral. L'adénovirus peut être utilisé pour introduire des protéines sériques via l'épithélium pulmonaire. L'hémophilie peut être traitée par introduction adénovirale du facteur VIII ou du facteur IX.
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