RU2018132517A - Aav-idua вектор для лечения ассоциированной с mps i слепоты - Google Patents

Aav-idua вектор для лечения ассоциированной с mps i слепоты Download PDF

Info

Publication number
RU2018132517A
RU2018132517A RU2018132517A RU2018132517A RU2018132517A RU 2018132517 A RU2018132517 A RU 2018132517A RU 2018132517 A RU2018132517 A RU 2018132517A RU 2018132517 A RU2018132517 A RU 2018132517A RU 2018132517 A RU2018132517 A RU 2018132517A
Authority
RU
Russia
Prior art keywords
aav
subject
cornea
particle
genome
Prior art date
Application number
RU2018132517A
Other languages
English (en)
Other versions
RU2018132517A3 (ru
Inventor
Мэттью Луис ХИРШ
Ричард Джуд САМУЛЬСКИ
Original Assignee
Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл
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 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл filed Critical Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл
Publication of RU2018132517A publication Critical patent/RU2018132517A/ru
Publication of RU2018132517A3 publication Critical patent/RU2018132517A3/ru

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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • C12N9/2411—Amylases
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
    • 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
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10044—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a 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
    • C12N2750/14141—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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Ophthalmology & Optometry (AREA)
  • Dermatology (AREA)
  • Immunology (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)
  • Enzymes And Modification Thereof (AREA)
  • Saccharide Compounds (AREA)

Claims (26)

1. Рекомбинантная нуклеиновая кислота, содержащая последовательность, кодирующую альфа-L-идуронидазу человека (IDUA), при этом нуклеотидная последовательность кодон-оптимизирована для экспрессии в клетках человека.
2. Рекомбинантная нуклеиновая кислота по п. 1, содержащая нуклеотидную последовательность, по меньшей мере на 90% идентичную SEQ ID NO:1.
3. Рекомбинантная нуклеиновая кислота по п. 1, содержащая нуклеотидную последовательность SEQ ID NO:1.
4. Геном вектора аденоассоциированного вируса (AAV), содержащий нуклеиновую кислоту по п. 1.
5. Геном вектора AAV по п. 4, в котором нуклеиновая кислота функционально связана с конститутивным промотором.
6. Клетка in vitro, содержащая геном вектора AAV по п. 4 или 5.
7. Клетка по п. 6, при этом векторный геном стабильно заключен в геноме клетки.
8. Частица AAV, содержащая геном вектора AAV по п. 4 или 5.
9. Способ получения рекомбинантной частицы AAV, содержащей капсид AAV, при этом способ предусматривает:
предоставление клетке in vitro кодирующих последовательностей Cap AAV и Rep AAV, генома вектора AAV по п. 4 или 5 и хелперных функций для генерирования продуктивной AAV-инфекции; и
создание возможности сборки рекомбинантной частицы AAV, содержащей капсид AAV, и заключение в капсид генома вектора AAV.
10. Частица AAV, получаемая посредством способа по п. 9.
11. Частица AAV по п. 8 или 10, при этом частица AAV представляет собой частицу AAV2, AAV8 или AAV9.
12. Частица AAV по п. 8 или 10, при этом частица AAV представляет собой химерную частицу AAV8/AAV9.
13. Фармацевтическая готовая форма, содержащая частицу AAV по любому одному из пп. 8 или 10-12 и фармацевтически приемлемый носитель.
14. Способ доставки IDUA в роговицу субъекта, предусматривающий введение в роговицу субъекта эффективного количества частицы AAV, которая экспрессирует IDUA, тем самым доставляя IDUA в роговицу субъекта.
15. Способ лечения или отсрочки начала ассоциированного с мукополисахаридозом I (MPS I) помутнения роговицы нуждающегося в этом субъекта, предусматривающий введение в роговицу субъекта терапевтически эффективного количества частицы AAV, которая экспрессирует IDUA, тем самым осуществляя лечение или отсрочку начала ассоциированного с MPS I помутнения роговицы у субъекта.
16. Способ доставки IDUA в роговицу in vitro или ex vivo, предусматривающий контакт роговицы с эффективным количеством частицы AAV, которая экспрессирует IDUA, тем самым доставляя IDUA в роговицу.
17. Способ по п. 16, дополнительно предусматривающий трансплантацию роговицы нуждающемуся в этом субъекту.
18. Способ по любому одному из пп. 14-17, при этом частица AAV представляет собой частицу AAV по любому одному из пп. 8 или 10-12 или фармацевтическую композицию по п. 13.
19. Способ по любому одному из пп. 14-18, в котором частицу AAV вводят в роговицу посредством интрастромальной инъекции.
20. Способ по любому одному из пп. 14-18, в котором частицу AAV вводят в роговицу местно.
21. Способ по любому одному из пп. 14-20, в котором субъектом является субъект-человек.
22. Способ по любому одному из пп. 14-21, в котором у субъекта диагностирован MPS I.
23. Способ по любому одному из пп. 14-22, в котором у субъекта не развилось помутнение роговицы.
24. Способ по любому одному из пп. 14-22, в котором у субъекта развилось помутнение роговицы.
RU2018132517A 2016-02-22 2017-02-22 Aav-idua вектор для лечения ассоциированной с mps i слепоты RU2018132517A (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662298126P 2016-02-22 2016-02-22
US62/298,126 2016-02-22
PCT/US2017/018829 WO2017147123A1 (en) 2016-02-22 2017-02-22 Aav-idua vector for treatment of mps i-associated blindness

Publications (2)

Publication Number Publication Date
RU2018132517A true RU2018132517A (ru) 2020-03-24
RU2018132517A3 RU2018132517A3 (ru) 2020-06-10

Family

ID=59685585

Family Applications (1)

Application Number Title Priority Date Filing Date
RU2018132517A RU2018132517A (ru) 2016-02-22 2017-02-22 Aav-idua вектор для лечения ассоциированной с mps i слепоты

Country Status (13)

Country Link
US (2) US11116850B2 (ru)
EP (1) EP3419639A4 (ru)
JP (2) JP7231922B2 (ru)
KR (1) KR20180109945A (ru)
CN (1) CN108697774A (ru)
AU (1) AU2017223465A1 (ru)
BR (1) BR112018017125A2 (ru)
CA (1) CA3011943A1 (ru)
HK (1) HK1255692A1 (ru)
IL (1) IL260643B2 (ru)
MX (1) MX2018009426A (ru)
RU (1) RU2018132517A (ru)
WO (1) WO2017147123A1 (ru)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL292830B2 (en) 2015-09-28 2023-06-01 Univ Florida Methods and compositions for stealth virus antibody vectors
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
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
MX2022004352A (es) 2019-10-17 2022-07-19 Stridebio Inc Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c.
US20220389457A1 (en) * 2019-10-23 2022-12-08 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN111718947B (zh) * 2020-06-18 2022-08-23 舒泰神（北京）生物制药股份有限公司 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
AU2022379625A1 (en) * 2021-10-27 2024-05-16 The University Of North Carolina At Chapel Hill Aav-idua vector for treatment of mps i

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US20030215422A1 (en) 1996-09-11 2003-11-20 John A. Chiorini Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
NZ528942A (en) 1998-09-22 2005-03-24 Univ Florida Methods for large-scale production of recombinant rAAV vectors
CA2349838C (en) 1998-11-05 2011-06-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
CN1311329A (zh) 2000-02-29 2001-09-05 复旦大学 一种新的多肽——人磷酸甘露糖异构酶16和编码这种多肽的多核苷酸
WO2001068888A2 (en) * 2000-03-14 2001-09-20 Neurologix, Inc. Production of chimeric capsid vectors
EP1290205B1 (en) 2000-06-01 2006-03-01 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
ES2437815T3 (es) * 2000-10-11 2014-01-14 Shire Human Genetic Therapies, Inc. ARN mensajero optimizado
CN103180445B (zh) * 2010-10-22 2018-02-16 库尔纳公司 通过抑制α‑L‑艾杜糖醛酸酶(IDUA)的天然反义转录物而治疗IDUA相关疾病
US8609088B2 (en) * 2011-05-10 2013-12-17 Regents Of The University Of Minnesota Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
US20130158103A1 (en) * 2011-11-23 2013-06-20 The Curators Of The University Of Missouri Method of Tissue-Selective Targeted Gene Transfer
SG11201507507PA (en) * 2013-03-15 2015-10-29 Univ Pennsylvania Compositions and methods for treating mpsi
US10077291B2 (en) 2013-03-15 2018-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV vectors
SG11201509419QA (en) 2013-05-15 2015-12-30 Univ Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
US20170178457A1 (en) 2014-07-08 2017-06-22 Jesus Franco Munoz Multi-player gaming machine
WO2016005514A1 (en) * 2014-07-10 2016-01-14 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of usher syndrome type 2

Also Published As

Publication number Publication date
HK1255692A1 (zh) 2019-08-23
JP2023011868A (ja) 2023-01-24
CA3011943A1 (en) 2017-08-31
EP3419639A4 (en) 2019-08-07
EP3419639A1 (en) 2019-01-02
US20190048363A1 (en) 2019-02-14
IL260643A (ru) 2018-09-20
JP2019511927A (ja) 2019-05-09
US11116850B2 (en) 2021-09-14
IL260643B2 (en) 2023-07-01
US20220047721A1 (en) 2022-02-17
BR112018017125A2 (pt) 2018-12-26
MX2018009426A (es) 2018-12-19
IL260643B1 (en) 2023-03-01
AU2017223465A1 (en) 2018-08-09
JP7231922B2 (ja) 2023-03-02
WO2017147123A1 (en) 2017-08-31
CN108697774A (zh) 2018-10-23
RU2018132517A3 (ru) 2020-06-10
KR20180109945A (ko) 2018-10-08

Similar Documents

Publication Publication Date Title
RU2018132517A (ru) Aav-idua вектор для лечения ассоциированной с mps i слепоты
AU2020200041B2 (en) Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer
JP7390693B2 (ja) 抗体回避性ウイルスベクターのための方法および組成物
US11702676B2 (en) Rational polyploid adeno-associated virus vectors for the treatment of disease
JP6831779B2 (ja) 修飾された第ｉｘ因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
Rapti et al. Adeno-associated viruses (AAV) and host immunity–A race between the hare and the hedgehog
US10011640B2 (en) Capsid-modified rAAV vector compositions and methods therefor
JP2023123765A (ja) 中枢神経系を標的化したａａｖベクター
JP6042825B2 (ja) 改変された形質導入プロファイルを有するウイルスベクターならびにその製造および使用の方法
CN107828820B (zh) 用于向神经系统细胞导入基因的腺相关病毒粒子
M Mitchell et al. AAV's anatomy: roadmap for optimizing vectors for translational success
JP2020533959A (ja) Ａａｖを送達するための組成物および方法
JP2018510620A5 (ru)
JP2014512171A5 (ru)
CA3054711A1 (en) Polyploid adeno-associated virus vectors and methods of making and using the same
RU2020137429A (ru) Лечение пигментного ретинита
JP2015532095A (ja) 希突起神経膠細胞を標的とするアデノ随伴ウイルスベクター
Vance et al. AAV biology, infectivity and therapeutic use from bench to clinic
HRP20201831T1 (hr) Optimizirani promoter rpe65 i kodirajuće sekvence
Zhan et al. Vectored immunotherapeutics for infectious diseases: Can rAAVs be the game changers for fighting transmissible pathogens?
JP2023154428A (ja) 合理的なポリプロイド性アデノ随伴ウイルスベクターならびにその製造法および使用法
KR20230152008A (ko) 단백질 m 유사체 및 융합 단백질 및 이들의 항체 기능 억제 용도
GB2562774A (en) Genetic construct
Yang et al. 526. Intravasuclar Delivery of RAAVRH. 8 Generates Widespreading Transduction of Neuronal and Glial Cell Types in the Adult Mouse Central Nervous System
US20230136849A1 (en) Capsid-modified raav vector compositions and methods therefor