CN105026554B - 用于治疗mps1的组合物和方法 - Google Patents

用于治疗mps1的组合物和方法 Download PDF

Info

Publication number
CN105026554B
CN105026554B CN201480010987.3A CN201480010987A CN105026554B CN 105026554 B CN105026554 B CN 105026554B CN 201480010987 A CN201480010987 A CN 201480010987A CN 105026554 B CN105026554 B CN 105026554B
Authority
CN
China
Prior art keywords
associated virus
vector
recombinant adeno
virus particle
recombinant
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
CN201480010987.3A
Other languages
English (en)
Chinese (zh)
Other versions
CN105026554A (zh
Inventor
J·M·威尔森
B·L·哥达
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.)
University of Pennsylvania Penn
Original Assignee
University of Pennsylvania Penn
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 University of Pennsylvania Penn filed Critical University of Pennsylvania Penn
Publication of CN105026554A publication Critical patent/CN105026554A/zh
Application granted granted Critical
Publication of CN105026554B publication Critical patent/CN105026554B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076L-Iduronidase (3.2.1.76)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
CN201480010987.3A 2013-03-15 2014-03-13 用于治疗mps1的组合物和方法 Active CN105026554B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361788724P 2013-03-15 2013-03-15
US61/788,724 2013-03-15
PCT/US2014/025509 WO2014151341A1 (en) 2013-03-15 2014-03-13 Compositions and methods for treating mpsi

Publications (2)

Publication Number Publication Date
CN105026554A CN105026554A (zh) 2015-11-04
CN105026554B true CN105026554B (zh) 2020-08-11

Family

ID=51580934

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201480010987.3A Active CN105026554B (zh) 2013-03-15 2014-03-13 用于治疗mps1的组合物和方法

Country Status (21)

Country Link
US (3) US10137176B2 (cg-RX-API-DMAC7.html)
EP (2) EP2984166B1 (cg-RX-API-DMAC7.html)
JP (4) JP6591956B2 (cg-RX-API-DMAC7.html)
KR (2) KR102346455B1 (cg-RX-API-DMAC7.html)
CN (1) CN105026554B (cg-RX-API-DMAC7.html)
AU (2) AU2014235096B2 (cg-RX-API-DMAC7.html)
BR (1) BR112015021036A2 (cg-RX-API-DMAC7.html)
CA (1) CA2901328C (cg-RX-API-DMAC7.html)
CY (1) CY1123169T1 (cg-RX-API-DMAC7.html)
DK (1) DK2984166T3 (cg-RX-API-DMAC7.html)
ES (2) ES2991471T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20201063T1 (cg-RX-API-DMAC7.html)
HU (1) HUE051373T2 (cg-RX-API-DMAC7.html)
LT (1) LT2984166T (cg-RX-API-DMAC7.html)
MX (2) MX386059B (cg-RX-API-DMAC7.html)
PL (1) PL2984166T3 (cg-RX-API-DMAC7.html)
PT (1) PT2984166T (cg-RX-API-DMAC7.html)
RU (1) RU2708318C2 (cg-RX-API-DMAC7.html)
SG (2) SG10201707319UA (cg-RX-API-DMAC7.html)
SI (1) SI2984166T1 (cg-RX-API-DMAC7.html)
WO (1) WO2014151341A1 (cg-RX-API-DMAC7.html)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6591956B2 (ja) * 2013-03-15 2019-10-16 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Mps1を治療するための組成物および方法
US11555059B2 (en) * 2014-04-25 2023-01-17 The Trustees Of The University Of Pennsylvania LDLR variants and their use in compositions for reducing cholesterol levels
IL259850B2 (en) * 2015-12-11 2023-11-01 Univ Pennsylvania Gene therapy for treating familial hypercholesterolemia
US11241506B2 (en) 2015-12-14 2022-02-08 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
WO2017136533A1 (en) * 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Methods for treating diagnosing, and monitoring treatment of mucopolysaccharidoses
US11116850B2 (en) * 2016-02-22 2021-09-14 The University Of North Carolina At Chapel Hill AAV-IDUA vector for treatment of MPS I-associated blindness
MA44874A (fr) 2016-04-15 2019-03-13 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type ii
IL322403A (en) * 2016-04-15 2025-09-01 Univ Pennsylvania Preparations for the treatment of wet age-related macular degeneration
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
KR20230161535A (ko) 2016-07-26 2023-11-27 바이오마린 파머수티컬 인크. 신규 아데노-관련 바이러스 캡시드 단백질
IL315358A (en) 2016-08-18 2024-11-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
WO2018126112A1 (en) * 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating phenylketonuria
WO2019010335A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
IL273427B2 (en) 2017-09-22 2025-04-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous polycythemia vera type II
CN107699590A (zh) * 2017-10-13 2018-02-16 成都中医药大学 一种制备重组人α‑L‑艾杜糖醛酸酶的方法
KR20200104864A (ko) 2017-11-30 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 뮤코다당류증 iiib형에 대한 유전자 요법
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
WO2019152843A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
JP7575273B2 (ja) 2018-04-16 2024-10-29 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法
AU2019265560A1 (en) 2018-05-09 2020-11-26 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
SG11202100139VA (en) 2018-07-17 2021-02-25 Neuromyon Inc Treatment of neuropathy with dna constructs expressing igf-1 isoforms
AU2020216480A1 (en) 2019-01-31 2021-09-09 Oregon Health & Science University Methods for using transcription-dependent directed evolution of AAV capsids
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
CA3183153A1 (en) 2020-06-17 2021-12-23 Christian HINDERER Compositions and methods for treatment of gene therapy patients
JP2023545433A (ja) 2020-10-09 2023-10-30 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ファブリー病の治療のための組成物及び方法
KR102590276B1 (ko) * 2021-01-04 2023-10-16 부산대학교 산학협력단 조직 특이적 발현을 위한 융합 프로모터 및 이의 용도
JP2024505257A (ja) 2021-02-01 2024-02-05 レジェンクスバイオ インコーポレーテッド 神経セロイドリポフスチン症の遺伝子治療
EP4148078B1 (de) 2021-09-08 2025-11-05 Covestro Deutschland AG Auf bernsteinsäure basierende polyesterpolyole
CN114181318B (zh) * 2021-11-08 2023-08-01 四川大学 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
CA3247109A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers
EP4504258A1 (en) 2022-04-06 2025-02-12 The Trustees of the University of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102869779A (zh) * 2010-03-29 2013-01-09 宾夕法尼亚大学托管会 药理学诱导的转基因消融系统

Family Cites Families (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6149909A (en) * 1995-06-23 2000-11-21 Women's And Children's Hospital Synthetic α-L-iduronidase and genetic sequences encoding same
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
JP2001500015A (ja) 1996-09-06 2001-01-09 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア T7ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
BR9910323A (pt) 1998-05-13 2001-01-30 Harbor Ucla Alfa-l-iduronidase recombinante, métodos para produção e purificação da mesma e métodos para tratamento de doenças ocasionadas deficiência da mesma
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
RU2196988C2 (ru) * 2000-04-19 2003-01-20 Государственный новосибирский областной клинический диагностический центр Способ диагностики мукополисахаридозов
MX346493B (es) 2001-11-13 2017-03-21 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
JP4810062B2 (ja) 2001-12-17 2011-11-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス(aav)血清型8の配列
WO2004055157A2 (en) 2002-08-13 2004-07-01 Whitley Chester B Methods of using vectors to treat metabolic disorders
US7155804B2 (en) 2003-09-17 2007-01-02 Moog Inc. Method of forming an electric motor
CN102199626B (zh) 2003-09-30 2015-06-24 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
EP2383346B1 (en) 2005-04-07 2014-10-08 The Trustees of the University of Pennsylvania Modified AAVrh.64 capsids, compositions containing same and uses thereof
EP1777906A1 (en) 2005-06-09 2007-04-25 Matsushita Electric Industrial Co., Ltd. Amplitude error compensating apparatus and orthogonality error compensating apparatus
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20090062144A1 (en) 2007-04-03 2009-03-05 Nancy Lan Guo Gene signature for prognosis and diagnosis of lung cancer
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
JP2014503173A (ja) * 2009-11-05 2014-02-13 プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ 制御された発現系
EP2333074A1 (en) * 2009-12-14 2011-06-15 Robert Steinfeld Substances and methods for the treatment of lysosmal storage diseases
CA2833908C (en) 2010-04-23 2021-02-09 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2012054723A2 (en) * 2010-10-22 2012-04-26 Opko Curna Llc Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
DK2675902T3 (da) 2011-02-17 2019-06-03 Univ Pennsylvania Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel
JP6072772B2 (ja) 2011-04-22 2017-02-01 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法
AU2013289206B2 (en) 2012-07-11 2018-08-09 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
JP6437443B2 (ja) 2012-11-21 2018-12-12 アールキューエックス ファーマシューティカルズ,インク. 大環状広域抗生物質
WO2014125647A1 (ja) 2013-02-18 2014-08-21 富士通オプティカルコンポーネンツ株式会社 光受信装置
JP6591956B2 (ja) 2013-03-15 2019-10-16 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Mps1を治療するための組成物および方法
EP3622821A1 (en) 2013-05-15 2020-03-18 Regents of the University of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
SG11201702073XA (en) 2014-09-24 2017-04-27 Hope City Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
EP3204503A1 (en) 2014-10-06 2017-08-16 Arthrogen BV Aav-based gene therapy
RU2751952C2 (ru) 2015-05-15 2021-07-21 Реджентс Оф Зэ Юниверсити Оф Миннесота Вектор на основе аденоассоциированного вируса для терапевтической доставки в центральную нервную систему
US10604747B2 (en) 2015-08-06 2020-03-31 Academia Sinica Engineered enzyme for enzyme replacement therapy
SG11201803218PA (en) 2015-10-23 2018-05-30 Univ Iowa Res Found Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
WO2017100676A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
EP3387118B1 (en) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
CA3010738A1 (en) 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
WO2019010335A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102869779A (zh) * 2010-03-29 2013-01-09 宾夕法尼亚大学托管会 药理学诱导的转基因消融系统

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
alpha-L-iduronidase precursor[Homo sapiens];登录号:NP_000194.2;《GenBank》;20130217;参见序列部分 *
Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery;Monica Cardone等;《Human Molecular Genetics》;20060227;第15卷(第7期);第1225-1236页,参见第1226页左栏第3段 *
Correction of Metabolic, Craniofacial, and Neurologic Abnormalities in MPS I Mice Treated at Birth with Adeno-associated Virus Vector Transducing the Human A-L-Iduronidase Gene;Seth D. Hartung等;《MOLECULAR THERAPY》;20040630;第9卷(第6期);第866-875页,参见摘要及第867页右栏第3-4段 *
Homo sapiens iduronidase,alpha-L-(IDUA),mRNA;登录号:NM_000203.3;《GenBank》;20130217;参见序列部分 *
IDUA Mutational Profiling of a Cohort of 102 European Patients with Mucopolysaccharidosis Type I: Identification and Characterization of 35 Novel α-L-iduronidase (IDUA) Alleles;Francesca Bertola1等;《HUMAN MUTATION》;20110331;第32卷;第2189-2210页,参见第2196页图1 *

Also Published As

Publication number Publication date
US10137176B2 (en) 2018-11-27
RU2708318C2 (ru) 2019-12-05
KR20150129678A (ko) 2015-11-20
JP6591956B2 (ja) 2019-10-16
AU2020203826A1 (en) 2020-07-02
BR112015021036A2 (pt) 2017-10-10
AU2014235096A1 (en) 2015-09-10
EP2984166A4 (en) 2016-09-07
CN105026554A (zh) 2015-11-04
HRP20201063T1 (hr) 2020-11-27
US20200397872A1 (en) 2020-12-24
DK2984166T3 (da) 2020-07-20
CA2901328C (en) 2023-08-01
JP7553527B2 (ja) 2024-09-18
LT2984166T (lt) 2020-08-10
ES2805355T3 (es) 2021-02-11
RU2015144234A (ru) 2017-04-24
CA2901328A1 (en) 2014-09-25
EP2984166A1 (en) 2016-02-17
JP2019134717A (ja) 2019-08-15
SI2984166T1 (sl) 2020-09-30
CY1123169T1 (el) 2021-10-29
AU2020203826B2 (en) 2022-09-15
SG11201507507PA (en) 2015-10-29
KR20220005599A (ko) 2022-01-13
JP7168621B2 (ja) 2022-11-09
US10792343B2 (en) 2020-10-06
EP3747998A9 (en) 2021-08-04
US20190111116A1 (en) 2019-04-18
MX2021010918A (es) 2021-10-01
ES2991471T3 (es) 2024-12-03
HK1221735A1 (en) 2017-06-09
EP2984166B1 (en) 2020-04-22
HUE051373T2 (hu) 2021-03-01
EP3747998A1 (en) 2020-12-09
MX2015012739A (es) 2016-02-19
SG10201707319UA (en) 2017-10-30
PT2984166T (pt) 2020-07-16
EP3747998B1 (en) 2024-07-24
JP2023011752A (ja) 2023-01-24
US20160000887A1 (en) 2016-01-07
JP2016512683A (ja) 2016-05-09
KR102581652B1 (ko) 2023-09-22
KR102346455B1 (ko) 2022-01-04
MX386059B (es) 2025-03-18
JP2020202863A (ja) 2020-12-24
AU2014235096B2 (en) 2020-03-19
PL2984166T3 (pl) 2021-01-11
WO2014151341A1 (en) 2014-09-25

Similar Documents

Publication Publication Date Title
JP7553527B2 (ja) Mps1を治療するための組成物および方法
KR20200052333A (ko) 아데노-관련 바이러스 변이체 캡시드 및 그 용도
JP2019533992A (ja) 酸性αグルコシダーゼ変異体及びその使用
JP2019506445A (ja) パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
CN111718947B (zh) 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途
CN110914419A (zh) 糖原贮积病iii的治疗
CN104936606A (zh) 糖原贮积病的基因疗法
US20230040603A1 (en) Compositions useful for treating gm1 gangliosidosis
US20230190966A1 (en) Compositions useful for treating gm1 gangliosidosis
HK40042501A (en) Compositions for treating mpsi
HK40042501B (en) Compositions for treating mpsi
US20240408157A1 (en) Compositions and methods for treatment of fabry disease
HK1221735B (en) Compositions for treating mpsi
EP4089171A1 (en) Recombinant tert-encoding viral genomes and vectors

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
GR01 Patent grant
GR01 Patent grant