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1. WO2020069339 - MUTANT REVERSE TETRACYCLINE TRANSACTIVATORS FOR EXPRESSION OF GENES

Publication Number WO/2020/069339
Publication Date 02.04.2020
International Application No. PCT/US2019/053492
International Filing Date 27.09.2019
IPC
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 15/63 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
C12N 15/90 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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
CPC
A61K 48/005
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
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
C12N 15/63
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
C12N 15/635
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
635Externally inducible repressor mediated regulation of gene expression, e.g. tetR inducible by tetracyline
C12N 2750/14143
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
2750ssDNA Viruses
00011ssDNA Viruses
14011Parvoviridae
14111Dependovirus, e.g. adenoassociated viruses
14141Use of virus, viral particle or viral elements as a vector
14143viral genome or elements thereof as genetic vector
C12N 2830/003
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
2830Vector systems having a special element relevant for transcription
001controllable enhancer/promoter combination
002inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
003tet inducible
Applicants
  • PRESIDENT AND FELLOWS OF HARVARD COLLEGE [US]/[US]
Inventors
  • SINCLAIR, David, A.
  • LU, Yuancheng
  • DAVIDSOHN, Noah, Justin
Agents
  • BAKER, C. Hunter
  • BALICH, Jason, W.
  • ATTISHA, Michael, J.
  • BAKER, C, Hunter
  • ALAM, Saad
Priority Data
62/738,89428.09.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) MUTANT REVERSE TETRACYCLINE TRANSACTIVATORS FOR EXPRESSION OF GENES
(FR) TRANSACTIVATEURS DE TÉTRACYCLINE INVERSES MUTANTS POUR L'EXPRESSION DE GÈNES
Abstract
(EN)
Provided herein are mutant reverse tetracycline transactivator (rtTA) proteins and engineered nucleic acids (e.g., viral vectors, including lentiviral vectors, adenoviral vectors, AAV vectors, herpes viral vectors, and retroviral vectors, and non-viral vectors, including RNA and plasmid DNA) that encode a mutant rtTA that are useful, for example, in regulating gene expression, inducing cellular reprogramming, tissue repair, tissue regeneration, organ regeneration, reversing aging, treating a disease (e.g., acute injuries, neurodegenerative disease, chronic diseases, proliferative diseases, cardiovascular diseases, genetic diseases, inflammatory diseases, autoimmunue diseases, neurological diseases, hematological diseases, painful conditions, psychiatric disorders, metabolic disorders, cancers, aging, age-related diseases, and diseases affecting any tissue in a subject), or any combination thereof. Also provided herein are recombinant viruses (e.g., lentiviruses, adenoviruses, alphaviruses, vaccinia viruses, retroviruses, herpes viruses, or A A Vs) comprising the engineered nucleic acids and methods of regulating (e.g., inhibiting or inducing) cellular reprogramming, tissue repair, tissue regeneration, or any combination thereof by administering an engineered nucleic acid or recombinant virus comprising the same in a cell, tissue or subject (e.g., a cell or tissue of a subject with a condition, which includes any disease (e.g., ocular disease), aging, neurodegenerative diseases, cancer, and age-related diseases) comprising administering a mutant rtTA and an inducible nucleic acid (e.g., an engineered nucleic acid, including an expression vector) encoding a transgene.
(FR)
L'invention concerne des protéines transactivateurs de tétracycline inverses (rtTA) mutants et des acides nucléiques modifiés (par exemple, des vecteurs viraux, comprenant des vecteurs lentiviraux, des vecteurs adénoviraux, des vecteurs AAV, des vecteurs herpès viraux, et des vecteurs rétroviraux, et des vecteurs non viraux, comprenant de l'ARN et de l'ADN plasmidique) qui codent pour un rtTA mutant qui sont utiles, par exemple, dans la régulation de l'expression génique, l'induction de la reprogrammation cellulaire, la réparation tissulaire, la régénération tissulaire, la régénération d'organe, l'inversion du vieillissement, le traitement d'une maladie (par exemple, de lésions aiguës, de maladies neurodégénératives, de maladies chroniques, de maladies prolifératives, de maladies cardiovasculaires, de maladies génétiques, de maladies inflammatoires, de maladies auto-immunes, de maladies neurologiques, de maladies hématologiques, d'affections douloureuses, de troubles psychiatriques, de troubles métaboliques, de cancers, du vieillissement, de maladies liées à l'âge et de maladies affectant n'importe quel tissu chez un sujet), ou toute combinaison de ceux-ci. L'invention concerne également des virus recombinants (par exemple, les lentivirus, les adénovirus, les alphavirus, les virus de la vaccine, les rétrovirus, les herpès virus ou les AAV) comprenant les acides nucléiques modifiés et des procédés de régulation (par exemple, inhibition ou induction) de la reprogrammation cellulaire, de la réparation tissulaire, de la régénération tissulaire, ou toute combinaison de celles-ci par l'administration d'un acide nucléique modifié ou d'un virus recombinant le comprenant dans une cellule, un tissu ou un sujet (par exemple, une cellule ou un tissu d'un sujet atteint d'une affection, qui comprend toute maladie (par exemple, une maladie oculaire), le vieillissement, les maladies neurodégénératives, le cancer et les maladies liées à l'âge) comprenant l'administration d'un rtTA mutant et d'un acide nucléique inductible (par exemple, un acide nucléique modifié, comprenant un vecteur d'expression) codant pour un transgène.
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