AU2019375914A1 - Synthetic immunomodulation with a CRISPR super-repressor in vivo - Google Patents

Synthetic immunomodulation with a CRISPR super-repressor in vivo Download PDF

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Publication number
AU2019375914A1
AU2019375914A1 AU2019375914A AU2019375914A AU2019375914A1 AU 2019375914 A1 AU2019375914 A1 AU 2019375914A1 AU 2019375914 A AU2019375914 A AU 2019375914A AU 2019375914 A AU2019375914 A AU 2019375914A AU 2019375914 A1 AU2019375914 A1 AU 2019375914A1
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Australia
Prior art keywords
repression
subject
myd88
crispr
synthetic
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Abandoned
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AU2019375914A
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English (en)
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Mo Reza Ebrahimkhani
Samira KIANI
Farzaneh MOGHADAM
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Arizona State University ASU
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Arizona State University ASU
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/635—Externally inducible repressor mediated regulation of gene expression, e.g. tetR inducible by tetracyline
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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
    • A61K48/0008—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 '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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2019375914A 2018-11-08 2019-11-07 Synthetic immunomodulation with a CRISPR super-repressor in vivo Abandoned AU2019375914A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862757679P 2018-11-08 2018-11-08
US62/757,679 2018-11-08
US201962899584P 2019-09-12 2019-09-12
US62/899,584 2019-09-12
PCT/US2019/060285 WO2020097344A1 (en) 2018-11-08 2019-11-07 Synthetic immunomodulation with a crispr super-repressor in vivo

Publications (1)

Publication Number Publication Date
AU2019375914A1 true AU2019375914A1 (en) 2021-06-03

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Application Number Title Priority Date Filing Date
AU2019375914A Abandoned AU2019375914A1 (en) 2018-11-08 2019-11-07 Synthetic immunomodulation with a CRISPR super-repressor in vivo

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US (2) US11884925B2 (https=)
EP (1) EP3877398A4 (https=)
JP (1) JP2022512974A (https=)
KR (1) KR20210102247A (https=)
CN (1) CN113330023A (https=)
AU (1) AU2019375914A1 (https=)
CA (1) CA3119302A1 (https=)
IL (1) IL283022A (https=)
SG (1) SG11202104802RA (https=)
WO (1) WO2020097344A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11773407B2 (en) 2017-06-26 2023-10-03 Arizona Board Of Regents On Behalf Of Arizona State University CRISPR logic circuits for safer and controllable gene therapies
US20200123567A1 (en) 2017-06-26 2020-04-23 Samira Kiani A universal platform to enhance crispr-based gene editing for in vivo therapies
WO2023288306A2 (en) * 2021-07-16 2023-01-19 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Targeting myd88 gene in vitro and in vivo
EP4479538A4 (en) * 2022-02-17 2026-01-28 Syntax Bio Inc CELL PROGRAMMING SYSTEMS AND ASSOCIATED PROCESSES
EP4711456A1 (en) * 2024-09-11 2026-03-18 GenCC GmbH & Co. KG Method for the manufacture of individualized crispr/cas complexes and individualized crispr/cas complexes

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US20030148986A1 (en) * 2001-12-17 2003-08-07 Cedars-Sinai Medical Center Methods for treating vascular disease by inhibiting myeloid differentiation factor 88
CN102166214B (zh) 2011-03-02 2012-12-12 华中科技大学同济医学院附属同济医院 氨基噻唑类MyD88特异性抑制剂在医学上的用途
US9228207B2 (en) * 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
JP6793547B2 (ja) * 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
CN106456801A (zh) * 2014-02-07 2017-02-22 Sbi生物技术有限公司 用于治疗肿瘤的抑制性寡核苷酸
WO2016049258A2 (en) * 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
WO2016094872A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
TWI906646B (zh) * 2015-06-18 2025-12-01 美商博得學院股份有限公司 降低脫靶效應的ｃｒｉｓｐｒ酶突變
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
NZ735250A (en) 2015-07-08 2020-08-28 Research & Business Found Sungkyunkwan Univ Pyrrolidine carboxamido derivatives and methods for preparing and using the same
WO2017068077A1 (en) 2015-10-20 2017-04-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
US11439692B2 (en) * 2017-05-17 2022-09-13 Modalis Therapeutics Corporation Method of treating diseases associated with MYD88 pathways using CRISPR-GNDM system
US11773407B2 (en) * 2017-06-26 2023-10-03 Arizona Board Of Regents On Behalf Of Arizona State University CRISPR logic circuits for safer and controllable gene therapies

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Publication number Publication date
SG11202104802RA (en) 2021-06-29
EP3877398A1 (en) 2021-09-15
IL283022A (en) 2021-06-30
CN113330023A (zh) 2021-08-31
KR20210102247A (ko) 2021-08-19
EP3877398A4 (en) 2022-08-17
US11884925B2 (en) 2024-01-30
WO2020097344A1 (en) 2020-05-14
US20210380990A1 (en) 2021-12-09
JP2022512974A (ja) 2022-02-07
CA3119302A1 (en) 2020-05-14
US20240117364A1 (en) 2024-04-11

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