BR112019000107A2 - composições e métodos com base em crispr / cas9 para o tratamento do câncer - Google Patents

composições e métodos com base em crispr / cas9 para o tratamento do câncer

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Publication number
BR112019000107A2
BR112019000107A2 BR112019000107-7A BR112019000107A BR112019000107A2 BR 112019000107 A2 BR112019000107 A2 BR 112019000107A2 BR 112019000107 A BR112019000107 A BR 112019000107A BR 112019000107 A2 BR112019000107 A2 BR 112019000107A2
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Brazil
Prior art keywords
crispr
methods
raav
cancer treatment
based compositions
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BR112019000107-7A
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English (en)
Inventor
Jaskula-Ranga Vinod
Zack Donald
Bunz Fred
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The Johns Hopkins University
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Publication date
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Publication of BR112019000107A2 publication Critical patent/BR112019000107A2/pt

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation 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
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
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    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Abstract

a presente invenção refere-se aos métodos para prevenir, inibir ou tratar câncer em um indivíduo. são também fornecidos na presente invenção os métodos para alterar a expressão de um ou mais produtos do gene em uma célula, tal como uma célula do câncer. tais métodos podem compreender o uso de um sistema de nuclease modificado, tal como repetições palindrómicas curtas intercaladas regularmente interconectadas (crispr) / crispr associadas (cas)9 (crispr-cas9) compreendendo um promotor h1 bidirecional e grnas dirigidos a oncogenes (raav-onco-crispr) ou genes supressores de tumor (raav-tsg) empacotados em uma partícula compacta de vírus adenoassociado (aav). tais métodos podem compreender a coadministração ou simultaneamente fornecer um adenovírus de empacotamento de vírus adenoassociado recombinante (ad-raavpack) com o sistema de nuclease.
BR112019000107-7A 2016-07-05 2017-07-05 composições e métodos com base em crispr / cas9 para o tratamento do câncer BR112019000107A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662358339P 2016-07-05 2016-07-05
US62/358,339 2016-07-05
PCT/US2017/040696 WO2018009525A1 (en) 2016-07-05 2017-07-05 Crispr/cas9-based compositions and methods for treating cancer

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BR112019000107A2 true BR112019000107A2 (pt) 2019-04-09

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US (1) US20200069818A1 (pt)
EP (1) EP3481431A4 (pt)
JP (1) JP2019520394A (pt)
KR (1) KR20190039115A (pt)
CN (1) CN109963598A (pt)
AU (1) AU2017292772A1 (pt)
BR (1) BR112019000107A2 (pt)
CA (1) CA3029908A1 (pt)
CL (1) CL2019000023A1 (pt)
EA (1) EA201990214A1 (pt)
IL (1) IL264034A (pt)
MX (1) MX2019000188A (pt)
SG (1) SG11201900028VA (pt)
WO (1) WO2018009525A1 (pt)

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CL2019000023A1 (es) 2019-06-21
AU2017292772A1 (en) 2019-02-21
JP2019520394A (ja) 2019-07-18
EP3481431A4 (en) 2020-01-01
WO2018009525A8 (en) 2019-02-07
MX2019000188A (es) 2019-06-20
SG11201900028VA (en) 2019-01-30
KR20190039115A (ko) 2019-04-10
EP3481431A1 (en) 2019-05-15
CN109963598A (zh) 2019-07-02
IL264034A (en) 2019-01-31
US20200069818A1 (en) 2020-03-05
WO2018009525A1 (en) 2018-01-11
CA3029908A1 (en) 2018-01-11
EA201990214A1 (ru) 2019-11-29

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