JP7624728B2 - Ｄｎａ結合ドメイントランスアクチベーター及びその使用 - Google Patents

Ｄｎａ結合ドメイントランスアクチベーター及びその使用 Download PDF

Info

Publication number
JP7624728B2
JP7624728B2 JP2021549631A JP2021549631A JP7624728B2 JP 7624728 B2 JP7624728 B2 JP 7624728B2 JP 2021549631 A JP2021549631 A JP 2021549631A JP 2021549631 A JP2021549631 A JP 2021549631A JP 7624728 B2 JP7624728 B2 JP 7624728B2
Authority
JP
Japan
Prior art keywords
seq
nucleic acid
fold
expression
dna
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
JP2021549631A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022521432A5 (https=
JP2022521432A (ja
Inventor
ミゲル セナ エステベス，
スコット エー． ウルフ，
Original Assignee
ユニバーシティ オブ マサチューセッツ
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 ユニバーシティ オブ マサチューセッツ filed Critical ユニバーシティ オブ マサチューセッツ
Publication of JP2022521432A publication Critical patent/JP2022521432A/ja
Publication of JP2022521432A5 publication Critical patent/JP2022521432A5/ja
Priority to JP2024184019A priority Critical patent/JP2025003531A/ja
Application granted granted Critical
Publication of JP7624728B2 publication Critical patent/JP7624728B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N15/11—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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Pain & Pain Management (AREA)
  • Epidemiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
JP2021549631A 2019-02-25 2020-02-24 Ｄｎａ結合ドメイントランスアクチベーター及びその使用 Active JP7624728B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024184019A JP2025003531A (ja) 2019-02-25 2024-10-18 Ｄｎａ結合ドメイントランスアクチベーター及びその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962810005P 2019-02-25 2019-02-25
US62/810,005 2019-02-25
PCT/US2020/019546 WO2020176426A1 (en) 2019-02-25 2020-02-24 Dna-binding domain transactivators and uses thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024184019A Division JP2025003531A (ja) 2019-02-25 2024-10-18 Ｄｎａ結合ドメイントランスアクチベーター及びその使用

Publications (3)

Publication Number Publication Date
JP2022521432A JP2022521432A (ja) 2022-04-07
JP2022521432A5 JP2022521432A5 (https=) 2023-03-03
JP7624728B2 true JP7624728B2 (ja) 2025-01-31

Family

ID=72238705

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021549631A Active JP7624728B2 (ja) 2019-02-25 2020-02-24 Ｄｎａ結合ドメイントランスアクチベーター及びその使用
JP2024184019A Pending JP2025003531A (ja) 2019-02-25 2024-10-18 Ｄｎａ結合ドメイントランスアクチベーター及びその使用

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024184019A Pending JP2025003531A (ja) 2019-02-25 2024-10-18 Ｄｎａ結合ドメイントランスアクチベーター及びその使用

Country Status (14)

Country Link
US (1) US12583902B2 (https=)
EP (1) EP3931213A4 (https=)
JP (2) JP7624728B2 (https=)
KR (1) KR20210132109A (https=)
CN (1) CN113710693B (https=)
AU (2) AU2020228028A1 (https=)
BR (1) BR112021016294A2 (https=)
CA (1) CA3129757A1 (https=)
CL (1) CL2021002226A1 (https=)
CO (1) CO2021011722A2 (https=)
IL (1) IL285687A (https=)
MX (1) MX2021010169A (https=)
SG (1) SG11202108568YA (https=)
WO (1) WO2020176426A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7624728B2 (ja) 2019-02-25 2025-01-31 ユニバーシティ オブ マサチューセッツ Ｄｎａ結合ドメイントランスアクチベーター及びその使用
WO2022020706A1 (en) * 2020-07-24 2022-01-27 University Of Massachusetts Dna-binding domain transactivators and uses thereof
WO2023042104A1 (en) * 2021-09-16 2023-03-23 Novartis Ag Novel transcription factors
JP2024545923A (ja) * 2021-12-30 2024-12-13 リーゲル セラピューティクス，インコーポレイティッド ナトリウム電位依存性チャネルアルファサブユニット１の発現を調節するための組成物、及びその使用
KR20250016657A (ko) * 2023-07-21 2025-02-04 한국화학연구원 dxCas9 및 CRP 유도체를 포함하는, 표적 유전자 발현 조절 시스템 및 이의 제조방법

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20180064827A1 (en) 2016-09-07 2018-03-08 Sangamo Therapeutics, Inc. Modulation of liver genes
JP2018513681A (ja) 2015-03-31 2018-05-31 エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. 細胞または生物のゲノムへのＤＮＡ配列の標的化組み込みのためのＣａｓ９レトロウイルスインテグラーゼおよびＣａｓ９レコンビナーゼ系
WO2018187363A1 (en) 2017-04-03 2018-10-11 Encoded Therapeutics, Inc. Tissue selective transgene expression

Family Cites Families (46)

* 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
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
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
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
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
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
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
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
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
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
ES2341926T3 (es) 1998-03-02 2010-06-29 Massachusetts Institute Of Technology Poliproteinas con dedos de cinc que tienen enlazadores mejorados.
US6794136B1 (en) * 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US20030082561A1 (en) 2000-07-21 2003-05-01 Takashi Sera Zinc finger domain recognition code and uses thereof
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
US20060182736A1 (en) 2003-06-10 2006-08-17 Kim Jin-Soo Transducible dna-binding proteins
WO2007061759A1 (en) 2005-11-18 2007-05-31 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Delayed expression vectors
AU2010273220B2 (en) 2009-07-13 2015-10-15 President And Fellows Of Harvard College Bifunctional stapled polypeptides and uses thereof
US9567573B2 (en) * 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
US9254311B2 (en) * 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
CA2954791C (en) * 2014-07-14 2025-11-18 The Regents Of The University Of California CRISPR/CAS TRANSCRIPTIONAL MODULATION
JP6853257B2 (ja) 2015-09-23 2021-03-31 サンガモ セラピューティクス， インコーポレイテッド Ｈｔｔリプレッサー及びその使用
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
GB201517227D0 (en) * 2015-09-29 2015-11-11 Univ Edinburgh Genetically-edited swine
IL310721B2 (en) * 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
GB2544270A (en) 2015-11-05 2017-05-17 Fundació Centre De Regulació Genòmica Nucleic acids, peptides and methods
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
US20190255106A1 (en) 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
JP2018088888A (ja) * 2016-12-06 2018-06-14 国立研究開発法人理化学研究所 Ｓｃｎ１ａ遺伝子の発現増強法
US11730828B2 (en) 2017-02-07 2023-08-22 The Regents Of The University Of California Gene therapy for haploinsufficiency
EP3717505A4 (en) 2017-12-01 2021-12-01 Encoded Therapeutics, Inc. GENENICALLY MODIFIED DNA-BINDING PROTEINS
CA3096407A1 (en) 2018-04-09 2019-10-17 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
WO2019224864A1 (ja) 2018-05-21 2019-11-28 国立研究開発法人理化学研究所 Ｓｃｎ１ａ遺伝子の発現増強法とそれによるドラベ症候群の治療法
WO2020097121A1 (en) 2018-11-05 2020-05-14 Allen Institute Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons
JP7624728B2 (ja) 2019-02-25 2025-01-31 ユニバーシティ オブ マサチューセッツ Ｄｎａ結合ドメイントランスアクチベーター及びその使用
PL3976798T3 (pl) 2019-05-29 2026-04-07 Encoded Therapeutics, Inc. Kompozycje i sposoby selektywnej regulacji genów
WO2022020706A1 (en) 2020-07-24 2022-01-27 University Of Massachusetts Dna-binding domain transactivators and uses thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018513681A (ja) 2015-03-31 2018-05-31 エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. 細胞または生物のゲノムへのＤＮＡ配列の標的化組み込みのためのＣａｓ９レトロウイルスインテグラーゼおよびＣａｓ９レコンビナーゼ系
US20180064827A1 (en) 2016-09-07 2018-03-08 Sangamo Therapeutics, Inc. Modulation of liver genes
WO2018187363A1 (en) 2017-04-03 2018-10-11 Encoded Therapeutics, Inc. Tissue selective transgene expression

Also Published As

Publication number Publication date
BR112021016294A2 (pt) 2021-10-13
US20220185862A1 (en) 2022-06-16
CA3129757A1 (en) 2020-09-03
CO2021011722A2 (es) 2021-12-10
CL2021002226A1 (es) 2022-04-29
US12583902B2 (en) 2026-03-24
CN113710693B (zh) 2025-07-15
AU2025234289A1 (en) 2025-10-16
CN113710693A (zh) 2021-11-26
WO2020176426A1 (en) 2020-09-03
EP3931213A1 (en) 2022-01-05
IL285687A (en) 2021-10-31
JP2025003531A (ja) 2025-01-09
EP3931213A4 (en) 2022-12-14
KR20210132109A (ko) 2021-11-03
MX2021010169A (es) 2021-12-10
SG11202108568YA (en) 2021-09-29
JP2022521432A (ja) 2022-04-07
AU2020228028A1 (en) 2021-09-30

Similar Documents

Publication Publication Date Title
JP7624728B2 (ja) Ｄｎａ結合ドメイントランスアクチベーター及びその使用
US20230073187A1 (en) Prostate targeting adeno-associated virus serotype vectors
JP7315475B2 (ja) 高活性制御エレメント
JP7535332B2 (ja) ミニ遺伝子療法
EP2497500B1 (en) Gene therapy for spinal muscular atrophy
JP2021521152A (ja) 抑制性ニューロンにおける電位開口型ナトリウムチャネルのレスキュー
CN111902539A (zh) 杂合调控元件
US20230279405A1 (en) Dna-binding domain transactivators and uses thereof
US20240309398A1 (en) Enhancers driving expression in motor neurons
US20240209354A1 (en) MULTIPLEX CRISPR/Cas9-MEDIATED TARGET GENE ACTIVATION SYSTEM
JP2024505570A (ja) 神経学的疾患及び疼痛を治療するためのエピジェネティック遺伝子調節
US20240207370A1 (en) Gene therapy for bcaa modulation in maple syrup urine disease (msud)
WO2024009280A1 (en) Integrated stress response inhibitors and methods of using the same
EA047753B1 (ru) Трансактиваторы днк-связывающего домена и их применение
WO2020210592A1 (en) Recombinant aav gene therapy for ngyl1 deficiency
US20240148830A1 (en) Treatment of Neurological Disorder Using NHR
WO2024129992A1 (en) Enhancers driving expression in motor neurons
WO2026039331A1 (en) Viral therapy for the treatment of mef2c haploinsufficiency syndrome
JP2025512470A (ja) アレキサンダー病における組換えＡＡＶ－ａｍｉＲＮＡによる遺伝子サイレンシング
CN120530197A (zh) 包括星形胶质细胞定向启动子构建体的合成核酸及其使用方法
BR112017027956B1 (pt) Vetores de vírus adenoassociado recombinante, uso e método de produção dos mesmos, e composição farmacêutica

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230222

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230222

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240112

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240401

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240618

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241018

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

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20241028

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: 20250109

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250114

R150 Certificate of patent or registration of utility model

Ref document number: 7624728

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150