CA2849241C - Construct - Google Patents

Construct Download PDF

Info

Publication number
CA2849241C
CA2849241C CA2849241A CA2849241A CA2849241C CA 2849241 C CA2849241 C CA 2849241C CA 2849241 A CA2849241 A CA 2849241A CA 2849241 A CA2849241 A CA 2849241A CA 2849241 C CA2849241 C CA 2849241C
Authority
CA
Canada
Prior art keywords
vector
aadc
dopamine
viral vector
sequence
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
CA2849241A
Other languages
English (en)
French (fr)
Other versions
CA2849241A1 (en
Inventor
Kyriacos Mitrophanous
Scott Ralph
Hannah Stewart
Alan Kingsman
Original Assignee
Oxford Biomedica UK Ltd
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 Oxford Biomedica UK Ltd filed Critical Oxford Biomedica UK Ltd
Priority to CA3052407A priority Critical patent/CA3052407C/en
Publication of CA2849241A1 publication Critical patent/CA2849241A1/en
Application granted granted Critical
Publication of CA2849241C publication Critical patent/CA2849241C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • C12N15/79—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
    • C12N15/867—Retroviral 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
    • 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
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/54—Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • 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
    • C12N9/88—Lyases (4.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P13/00—Preparation of nitrogen-containing organic compounds
    • C12P13/001—Amines; Imines
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16002—Tyrosine 3-monooxygenase (1.14.16.2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04016—GTP cyclohydrolase I (3.5.4.16)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01028—Aromatic-L-amino-acid decarboxylase (4.1.1.28), i.e. tryptophane-decarboxylase
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Psychiatry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA2849241A 2011-10-28 2012-10-26 Construct Active CA2849241C (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA3052407A CA3052407C (en) 2011-10-28 2012-10-26 Construct

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201161552581P 2011-10-28 2011-10-28
GB1118636.8 2011-10-28
US61/552,581 2011-10-28
GBGB1118636.8A GB201118636D0 (en) 2011-10-28 2011-10-28 Nucleotide sequence
PCT/GB2012/052668 WO2013061076A1 (en) 2011-10-28 2012-10-26 Construct

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CA3052407A Division CA3052407C (en) 2011-10-28 2012-10-26 Construct

Publications (2)

Publication Number Publication Date
CA2849241A1 CA2849241A1 (en) 2013-05-02
CA2849241C true CA2849241C (en) 2019-12-31

Family

ID=45373567

Family Applications (2)

Application Number Title Priority Date Filing Date
CA2849241A Active CA2849241C (en) 2011-10-28 2012-10-26 Construct
CA3052407A Active CA3052407C (en) 2011-10-28 2012-10-26 Construct

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA3052407A Active CA3052407C (en) 2011-10-28 2012-10-26 Construct

Country Status (11)

Country Link
US (1) US11279954B2 (https=)
EP (2) EP2771471B1 (https=)
JP (1) JP6296987B2 (https=)
CN (2) CN107523581B (https=)
AU (1) AU2012328166B2 (https=)
CA (2) CA2849241C (https=)
DK (2) DK2771471T3 (https=)
ES (2) ES2628419T3 (https=)
GB (1) GB201118636D0 (https=)
PL (2) PL2771471T3 (https=)
WO (1) WO2013061076A1 (https=)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201322798D0 (en) 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
KR20180034467A (ko) * 2015-08-03 2018-04-04 마이오도파 리미티드 L-dopa의 전신 합성 및 조절
GB2562774A (en) * 2017-05-25 2018-11-28 Mcdonald Michael Genetic construct
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
CN108841868A (zh) * 2018-05-31 2018-11-20 康霖生物科技（杭州）有限公司 一种用于中枢神经系统疾病治疗的基因序列构建体
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US20220175962A1 (en) * 2019-03-10 2022-06-09 Sio Gene Therapies Inc. Gene therapy compositions and methods for treating parkinson's disease
CN109971729B (zh) * 2019-04-19 2021-07-16 上海信致医药科技有限公司 一种酶组合物
GB2587319A (en) 2019-08-12 2021-03-31 Healing Genes Llc Genetic construct
CN118360338A (zh) * 2020-03-13 2024-07-19 康霖生物科技（杭州）有限公司 一种核酸构建体
GB2601752A (en) 2020-12-08 2022-06-15 Maavrx Ltd Expression vector
JP2024546964A (ja) 2021-12-17 2024-12-26 インペリアル カレッジ イノベイションズ リミテッド Ｒｎａ構築物
CN114317610B (zh) * 2022-01-27 2024-07-02 上海本导基因技术有限公司 一种适用于帕金森疾病基因治疗的慢病毒载体
WO2023202637A1 (en) 2022-04-19 2023-10-26 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating neurodegenerative disorders
CN117106825B (zh) * 2023-08-28 2024-11-01 康霖生物科技（杭州）有限公司 用于治疗帕金森病的基因治疗载体及其用途
CN118389675B (zh) * 2024-05-27 2025-06-27 南方医科大学珠江医院 芳香族氨基酸脱羧酶作为标志物在肝性脑病的检测产品中的应用

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
DE4006630A1 (de) 1990-03-03 1991-09-12 Behringwerke Ag Humane monoklonale antikoerper gegen tollwutviren, ihre herstellung und verwendung
US5512421A (en) 1991-02-19 1996-04-30 The Regents Of The University Of California Generation, concentration and efficient transfer of VSV-G pseudotyped retroviral vectors
CA2111234C (en) 1991-06-21 2002-11-26 Manoj Chalam Method for increasing rainout from autorefrigerated vaporous cloud containing hydrofluoric acid
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
AU1073797A (en) * 1995-11-14 1997-06-05 Somatix Therapy Corporation Joint expression of gtp cyclohydrolase and tyrosine hydroxylase
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
WO1999061639A2 (en) 1998-05-22 1999-12-02 Oxford Biomedica (Uk) Limited Retroviral delivery system
PL332875A1 (en) 1996-10-17 1999-10-25 Oxford Biomedica Ltd Retroviral vectors
GB9622500D0 (en) * 1996-10-29 1997-01-08 Oxford Biomedica Ltd Therapeutic gene
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
GB9803351D0 (en) 1998-02-17 1998-04-15 Oxford Biomedica Ltd Anti-viral vectors
GB9904905D0 (en) 1999-03-03 1999-04-28 Oxford Biomedica Ltd Cells
MXPA02000483A (es) 1999-07-13 2002-07-02 Pellicer Carlos F Panel para la construccion e instalacion para su fabricacion.
GB0009760D0 (en) 2000-04-19 2000-06-07 Oxford Biomedica Ltd Method
GB0024550D0 (https=) * 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1504108B1 (en) 2002-02-01 2013-04-17 Oxford Biomedica (UK) Limited Lentiviral vector
SE0302509D0 (sv) 2003-09-19 2003-09-19 Amersham Biosciences Ab Matrix for separation of polyethers and method of separation
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
GB0526210D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Vectors
US20090214478A1 (en) 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
PL2307551T3 (pl) 2008-06-18 2017-07-31 Oxford Biomedica (Uk) Limited Oczyszczanie wektorów retrowirusowych
JP5559185B2 (ja) * 2008-11-11 2014-07-23 オックスフォード バイオメディカ（ユーケー）リミテッド 方法
CN102946907A (zh) * 2010-05-28 2013-02-27 牛津生物医学（英国）有限公司 慢病毒载体向脑的递送

Also Published As

Publication number Publication date
DK3219801T3 (da) 2021-02-22
ES2628419T3 (es) 2017-08-02
EP2771471A1 (en) 2014-09-03
CN107523581A (zh) 2017-12-29
DK2771471T3 (en) 2017-08-21
PL3219801T3 (pl) 2021-06-14
EP2771471B1 (en) 2017-05-03
EP3219801A1 (en) 2017-09-20
CA3052407C (en) 2020-03-31
GB201118636D0 (en) 2011-12-07
EP3219801B1 (en) 2020-12-16
CN107523581B (zh) 2021-08-17
PL2771471T3 (pl) 2017-10-31
ES2857003T3 (es) 2021-09-28
JP2014532402A (ja) 2014-12-08
JP6296987B2 (ja) 2018-03-20
AU2012328166A1 (en) 2014-06-12
WO2013061076A1 (en) 2013-05-02
AU2012328166B2 (en) 2018-03-08
US20200040361A1 (en) 2020-02-06
US11279954B2 (en) 2022-03-22
CA2849241A1 (en) 2013-05-02
CN104011213A (zh) 2014-08-27
CN104011213B (zh) 2017-10-03
CA3052407A1 (en) 2013-05-02

Similar Documents

Publication Publication Date Title
US11279954B2 (en) Catecholamine enzyme fusions
JP7634507B2 (ja) 操作されたｄｎａ結合タンパク質
AU2020282352B2 (en) Compositions and methods for selective gene regulation
EP1337655B1 (en) Lentiviral vectors for the treatment of neurodegenerative diseases
US12357707B2 (en) Compositions and methods for treating laminopathies
KR20240025507A (ko) 미성숙 종결 코돈-매개 장애를 치료하기 위한 방법 및 조성물
US10400252B2 (en) Catecholamine enzyme fusions
JP6698114B2 (ja) 構築物
US20220175962A1 (en) Gene therapy compositions and methods for treating parkinson's disease

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20170803

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 12TH ANNIV.) - STANDARD

Year of fee payment: 12

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20241018

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20241018

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20241018

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 13TH ANNIV.) - STANDARD

Year of fee payment: 13

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20251011

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20251011