JP7658580B2 - アルファヘルペスウイルスプロモーター配列を含む組換え核酸 - Google Patents

アルファヘルペスウイルスプロモーター配列を含む組換え核酸 Download PDF

Info

Publication number
JP7658580B2
JP7658580B2 JP2021545307A JP2021545307A JP7658580B2 JP 7658580 B2 JP7658580 B2 JP 7658580B2 JP 2021545307 A JP2021545307 A JP 2021545307A JP 2021545307 A JP2021545307 A JP 2021545307A JP 7658580 B2 JP7658580 B2 JP 7658580B2
Authority
JP
Japan
Prior art keywords
seq
nucleic acid
cell
promoter
item
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.)
Active
Application number
JP2021545307A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022518867A (ja
JP2022518867A5 (enExample
Inventor
エンゲル，エステバン
マトゥラーナ，カローラ
エンキスト，リン
Original Assignee
ザ、トラスティーズ オブ プリンストン ユニバーシティ
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 ザ、トラスティーズ オブ プリンストン ユニバーシティ filed Critical ザ、トラスティーズ オブ プリンストン ユニバーシティ
Publication of JP2022518867A publication Critical patent/JP2022518867A/ja
Publication of JP2022518867A5 publication Critical patent/JP2022518867A5/ja
Priority to JP2024213389A priority Critical patent/JP2025026584A/ja
Application granted granted Critical
Publication of JP7658580B2 publication Critical patent/JP7658580B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16711—Varicellovirus, e.g. human herpesvirus 3, Varicella Zoster, pseudorabies
    • C12N2710/16722—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6897—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Analytical Chemistry (AREA)
  • Immunology (AREA)
JP2021545307A 2019-02-05 2020-02-05 アルファヘルペスウイルスプロモーター配列を含む組換え核酸 Active JP7658580B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024213389A JP2025026584A (ja) 2019-02-05 2024-12-06 アルファヘルペスウイルスプロモーター配列を含む組換え核酸

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962801524P 2019-02-05 2019-02-05
US62/801,524 2019-02-05
US201962950848P 2019-12-19 2019-12-19
US62/950,848 2019-12-19
PCT/US2020/016787 WO2020163475A1 (en) 2019-02-05 2020-02-05 Recombinant nucleic acids containing alphaherpesvirus promoter sequences

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024213389A Division JP2025026584A (ja) 2019-02-05 2024-12-06 アルファヘルペスウイルスプロモーター配列を含む組換え核酸

Publications (3)

Publication Number Publication Date
JP2022518867A JP2022518867A (ja) 2022-03-16
JP2022518867A5 JP2022518867A5 (enExample) 2023-02-13
JP7658580B2 true JP7658580B2 (ja) 2025-04-08

Family

ID=71947095

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021545307A Active JP7658580B2 (ja) 2019-02-05 2020-02-05 アルファヘルペスウイルスプロモーター配列を含む組換え核酸
JP2024213389A Pending JP2025026584A (ja) 2019-02-05 2024-12-06 アルファヘルペスウイルスプロモーター配列を含む組換え核酸

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024213389A Pending JP2025026584A (ja) 2019-02-05 2024-12-06 アルファヘルペスウイルスプロモーター配列を含む組換え核酸

Country Status (11)

Country Link
US (1) US12551574B2 (enExample)
EP (1) EP3921431A4 (enExample)
JP (2) JP7658580B2 (enExample)
KR (1) KR20210125014A (enExample)
CN (1) CN113383082A (enExample)
AU (1) AU2020218337A1 (enExample)
BR (1) BR112021015281A2 (enExample)
CA (1) CA3127019A1 (enExample)
MX (1) MX2021009161A (enExample)
SG (1) SG11202108111WA (enExample)
WO (1) WO2020163475A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7658580B2 (ja) 2019-02-05 2025-04-08 ザ、トラスティーズ オブ プリンストン ユニバーシティ アルファヘルペスウイルスプロモーター配列を含む組換え核酸
US20250025575A1 (en) * 2021-11-23 2025-01-23 The Trustees Of Princeton University Compositions and Methods for Cell-Specific Expression of Target Genes
WO2026032009A1 (en) * 2024-08-06 2026-02-12 Chinese Institute For Brain Research, Beijing (Cibr) A microglia-specific regulatory element for aav-mediated transgene manipulation

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5352596A (en) * 1992-09-11 1994-10-04 The United States Of America As Represented By The Secretary Of Agriculture Pseudorabies virus deletion mutants involving the EPO and LLT genes
US7341847B2 (en) * 2003-04-02 2008-03-11 Agency For Science, Technology And Research Promoter construct for gene expression in neuronal cells
WO2005080581A2 (en) * 2004-02-17 2005-09-01 University Of Florida Research Foundation, Inc. Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
JP7090548B2 (ja) 2016-01-29 2022-06-24 ジリオン バイオテック ゲーエムベーハー Ａａｖ系条件的発現系
JP7658580B2 (ja) 2019-02-05 2025-04-08 ザ、トラスティーズ オブ プリンストン ユニバーシティ アルファヘルペスウイルスプロモーター配列を含む組換え核酸
US20250025575A1 (en) 2021-11-23 2025-01-23 The Trustees Of Princeton University Compositions and Methods for Cell-Specific Expression of Target Genes

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
Arch Virol，1999年，144，pp.381-391
Database GenBank [online], Accession No. L13855，1994年02月25日，https://www.ncbi.nlm.nih.gov/nuccore/L13855
Database GenBank [online], Accession No.JF797219，2011年11月02日，https://www.ncbi.nlm.nih.gov/nuccore/JF797219.1
Drug Delivery System，2007年，22-6，pp. 643-650
HUMAN GENE THERAPY，2008年，19，pp.648-654
JOURNAL OF VIROLOGY，1993年，Vol.67, No.10，p.5955-5961
Promega Technical Manual pGL2 Luciferase Reporter Vectors，2007年，Page 4

Also Published As

Publication number Publication date
EP3921431A1 (en) 2021-12-15
CA3127019A1 (en) 2020-08-13
WO2020163475A1 (en) 2020-08-13
JP2025026584A (ja) 2025-02-21
JP2022518867A (ja) 2022-03-16
BR112021015281A2 (pt) 2021-10-05
EP3921431A4 (en) 2022-12-14
CN113383082A (zh) 2021-09-10
KR20210125014A (ko) 2021-10-15
MX2021009161A (es) 2021-09-10
US12551574B2 (en) 2026-02-17
SG11202108111WA (en) 2021-08-30
US20220378941A1 (en) 2022-12-01
AU2020218337A1 (en) 2021-08-26

Similar Documents

Publication Publication Date Title
JP2025026584A (ja) アルファヘルペスウイルスプロモーター配列を含む組換え核酸
CN109641939B (zh) 变体腺伴随病毒及使用方法
JP7066619B2 (ja) アデノ随伴ウイルス（ａａｖ）を誘導するための標的指向性ペプチド
JP2023093461A (ja) ハンチントン病治療組成物および方法
EP4334332A1 (en) Recombinant aavs for delivery to central nervous system and brain vasculature
TW201905200A (zh) 調節多核苷酸
JP2018512125A (ja) 多重ベクターシステム及びその使用
JP2021534814A (ja) アルファ−シヌクレインに対するバリアントＲＮＡｉ
KR20230051208A (ko) 신규 aav 캡시드 및 이를 함유하는 조성물
JP2024109708A (ja) 非症候性感音性聴力喪失の治療方法
Maturana et al. Small alphaherpesvirus latency-associated promoters drive efficient and long-term transgene expression in the CNS
AU2023367210A1 (en) Targets for receptor-mediated control of therapeutic biodistribution and efficacy
US20240309398A1 (en) Enhancers driving expression in motor neurons
TW202421789A (zh) 用於去靶向背根神經節及／或肝臟中之基因表現的元件
US20230279405A1 (en) Dna-binding domain transactivators and uses thereof
JP2024507732A (ja) ハイッブリッドａａｖ－アネロベクター
US20250025575A1 (en) Compositions and Methods for Cell-Specific Expression of Target Genes
Galvan et al. MiniPromoters Ple384 (TH) and Ple388 (PITX3) for targeting midbrain dopaminergic neurons in mice and monkeys
TWI883636B (zh) 含有星狀細胞導向啟動子構築體之合成核酸及其使用方法
US20240279679A1 (en) Compositions and methods for treatment of spinal muscular atrophy
WO2025235689A2 (en) Peptides targeting alpl for blood-brain barrier crossing
WO2024129992A1 (en) Enhancers driving expression in motor neurons
EA047753B1 (ru) Трансактиваторы днк-связывающего домена и их применение

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230203

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230203

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20240112

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240205

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240502

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240624

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240807

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241206

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20241209

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20241227

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20250307

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250319

R150 Certificate of patent or registration of utility model

Ref document number: 7658580

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150