CA3113470A1 - Mutant reverse tetracycline transactivators for expression of genes - Google Patents

Mutant reverse tetracycline transactivators for expression of genes Download PDF

Info

Publication number
CA3113470A1
CA3113470A1 CA3113470A CA3113470A CA3113470A1 CA 3113470 A1 CA3113470 A1 CA 3113470A1 CA 3113470 A CA3113470 A CA 3113470A CA 3113470 A CA3113470 A CA 3113470A CA 3113470 A1 CA3113470 A1 CA 3113470A1
Authority
CA
Canada
Prior art keywords
nucleic acid
sequence
engineered nucleic
seq
promoter
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3113470A
Other languages
English (en)
French (fr)
Inventor
David A. Sinclair
Yuancheng Lu
Noah Justin DAVIDSOHN
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Harvard University
Original Assignee
Harvard University
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Harvard University filed Critical Harvard University
Publication of CA3113470A1 publication Critical patent/CA3113470A1/en
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
CA3113470A 2018-09-28 2019-09-27 Mutant reverse tetracycline transactivators for expression of genes Pending CA3113470A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862738894P 2018-09-28 2018-09-28
US62/738,894 2018-09-28
PCT/US2019/053492 WO2020069339A1 (en) 2018-09-28 2019-09-27 Mutant reverse tetracycline transactivators for expression of genes

Publications (1)

Publication Number Publication Date
CA3113470A1 true CA3113470A1 (en) 2020-04-02

Family

ID=68242854

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3113470A Pending CA3113470A1 (en) 2018-09-28 2019-09-27 Mutant reverse tetracycline transactivators for expression of genes

Country Status (6)

Country Link
US (1) US20210403923A1 (https=)
EP (1) EP3856260A1 (https=)
JP (1) JP7589997B2 (https=)
CN (1) CN113164625B (https=)
CA (1) CA3113470A1 (https=)
WO (1) WO2020069339A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12274733B2 (en) 2018-09-28 2025-04-15 President And Fellows Of Harvard College Cellular reprogramming to reverse aging and promote organ and tissue regeneration
MX2021003663A (es) 2018-09-28 2021-05-28 Harvard College Reprogramacion celular para revertir el envejecimiento y promover la regeneracion de organos y tejidos.
WO2021091935A1 (en) * 2019-11-05 2021-05-14 Lonza Walkersville, Inc. Allogeneic t-cells and methods for production thereof
GB202103327D0 (en) * 2021-03-10 2021-04-21 Babraham Inst Novel method
US12467917B2 (en) 2021-03-16 2025-11-11 California Institute Of Technology Reconstructing human early embryogenesis in vitro with pluripotent stem cells
EP4355882A2 (en) * 2021-06-15 2024-04-24 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
JP2025511883A (ja) * 2022-04-06 2025-04-16 プレジデント アンド フェローズ オブ ハーバード カレッジ 中枢神経系の加齢を逆行させること
CN119546324A (zh) * 2022-07-12 2025-02-28 南京瑞初医药有限公司 组合物及其使用方法
US20260035707A1 (en) * 2022-07-26 2026-02-05 Modernatx, Inc. Engineered polynucleotides for temporal control of expression
CN116004789B (zh) * 2022-08-01 2024-06-04 湖南家辉生物技术有限公司 一种Seckel综合征致病基因CEP152突变位点的应用及其诊断试剂
US12252518B2 (en) 2023-01-06 2025-03-18 Life Biosciences, Inc. Methods of treating non-arteritic anterior ischemic optic neuropathy

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
AU746850B2 (en) * 1994-07-01 2002-05-02 Tet Systems Holding Gmbh & Co. Kg Tetracycline-regulated transcriptional modulaters
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
DE19851415B4 (de) * 1998-11-07 2010-11-04 Wolfgang Hillen Mutanter Transaktivator
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
US20030186281A1 (en) * 2001-12-21 2003-10-02 Wolfgang Hillen Modified tetracycline repressor protein compositions and methods of use
GB0327384D0 (en) * 2003-11-25 2003-12-31 Queen Mary & Westfield College Gene therapy
JP5155176B2 (ja) * 2005-11-17 2013-02-27 ティーイーティー システムズ ゲーエムベーハー ウント ツェーオー． カーゲー 誘導発現系
WO2008051854A2 (en) * 2006-10-20 2008-05-02 Trustees Of Boston University A tunable genetic switch for regulating gene expression
US8080647B2 (en) * 2006-11-22 2011-12-20 Pioneer Hi Bred International Inc Tetracycline repressor and uses thereof
CN101302553A (zh) * 2008-05-30 2008-11-12 广州华峰生物科技有限公司 基于环介导等温扩增技术的结核分歧杆菌基因快速诊断试剂盒及其检测方法
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP2643459B1 (en) * 2010-11-24 2017-09-27 Takara Bio USA, Inc. Inducible expression system transcription modulators comprising a distributed protein transduction domain and methods for using the same
EP2710130B1 (en) * 2011-05-19 2022-06-29 Fundación Pública Andaluza Progreso Y Salud Highly inducible dual-promoter lentiviral tet-on system
CN102816793B (zh) * 2012-08-22 2014-09-10 陕西师范大学 单载体双向启动的Tet-on诱导表达系统及其构建方法和应用
DK2898076T3 (en) * 2012-09-19 2018-06-14 Dsm Ip Assets Bv METHOD OF CELL MODIFICATION USING ESSENTIAL GENES AS MARKERS AND EVEN RECYCLING THESE
KR20150087321A (ko) * 2012-11-21 2015-07-29 메르츠 파마 게엠베하 운트 코. 카가아 보툴리눔 신경독소 생물학적 활성의 결정을 위한 수단 및 방법
WO2014152607A2 (en) * 2013-03-14 2014-09-25 University Of Massachusetts Dosage compensating transgenes and cells
EP3298141A4 (en) * 2015-05-17 2019-01-09 The Medical Research, Infrastructure, And Health Services Fund Of The Tel Aviv Medical Center COMPOSITIONS AND METHOD FOR THE TREATMENT OF CANCER
AU2016315703A1 (en) * 2015-08-31 2018-03-08 The Trustees Of The University Of Pennsylvania Chimeric AAV-anti-VEGF for treating cancer in canines
WO2019023680A1 (en) * 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
WO2019099552A1 (en) * 2017-11-14 2019-05-23 The J. David Gladstone Institutes Methods of generating retinal pigment epithelium (rpe)

Also Published As

Publication number Publication date
US20210403923A1 (en) 2021-12-30
CN113164625B (zh) 2026-04-14
JP2022501060A (ja) 2022-01-06
WO2020069339A8 (en) 2020-05-07
WO2020069339A1 (en) 2020-04-02
JP7589997B2 (ja) 2024-11-26
EP3856260A1 (en) 2021-08-04
CN113164625A (zh) 2021-07-23

Similar Documents

Publication Publication Date Title
US20210403923A1 (en) Mutant reverse tetracycline transactivators for expression of genes
US12409207B2 (en) Cellular reprogramming to reverse aging and promote organ and tissue regeneration
US12274733B2 (en) Cellular reprogramming to reverse aging and promote organ and tissue regeneration
US20250325628A1 (en) Reversing aging of the central nervous system
US20250109398A1 (en) Compositions for treating neurological disease
Cubinkova et al. Next generation tau models in Alzheimer's disease research-virus based gene delivery systems
JP7807393B2 (ja) 調節核酸配列
US20250009906A1 (en) Nociceptor-specific gene regulatory elements for the treatment of pain
HK40088903A (zh) 调节性核酸序列

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220826

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 5TH ANNIV.) - STANDARD

Year of fee payment: 5

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20240920

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20240920

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20240920

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20250205

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20250604

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250605

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20250716

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20250716

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 6TH ANNIV.) - STANDARD

Year of fee payment: 6

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250919

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250919