[go: up one dir, main page]

EA201991911A1 - Способы и композиции для переноса генов по сосудистой сети - Google Patents

Способы и композиции для переноса генов по сосудистой сети

Info

Publication number
EA201991911A1
EA201991911A1 EA201991911A EA201991911A EA201991911A1 EA 201991911 A1 EA201991911 A1 EA 201991911A1 EA 201991911 A EA201991911 A EA 201991911A EA 201991911 A EA201991911 A EA 201991911A EA 201991911 A1 EA201991911 A1 EA 201991911A1
Authority
EA
Eurasian Patent Office
Prior art keywords
genes
compositions
transfer
methods
viral
Prior art date
Application number
EA201991911A
Other languages
English (en)
Other versions
EA038695B1 (ru
Inventor
Аравинд Асокан
Гиридхар Мурлидхаран
Блейк Олбрайт
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 EA201991911A1 publication Critical patent/EA201991911A1/ru
Publication of EA038695B1 publication Critical patent/EA038695B1/ru

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • 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
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14122—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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

Изобретение относится к капсидным белкам AAV, содержащим модификацию в аминокислотной последовательности, и капсидам вирусов и вирусным векторам, содержащим модифицированный капсидный белок AAV. Изобретение также относится к способам введения вирусных векторов и капсидов вирусов согласно раскрытию в клетку или пациенту in vivo.
EA201991911A 2017-02-15 2018-02-15 Способы и композиции для переноса генов по сосудистой сети EA038695B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762459286P 2017-02-15 2017-02-15
PCT/US2018/018381 WO2018152333A1 (en) 2017-02-15 2018-02-15 Methods and compositions for gene transfer across the vasculature

Publications (2)

Publication Number Publication Date
EA201991911A1 true EA201991911A1 (ru) 2020-02-20
EA038695B1 EA038695B1 (ru) 2021-10-06

Family

ID=63169716

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201991911A EA038695B1 (ru) 2017-02-15 2018-02-15 Способы и композиции для переноса генов по сосудистой сети

Country Status (13)

Country Link
US (2) US11905312B2 (ru)
EP (1) EP3583220A4 (ru)
JP (2) JP7291397B2 (ru)
KR (1) KR20190117571A (ru)
CN (2) CN110520536B (ru)
AU (1) AU2018221738B2 (ru)
BR (1) BR112019016769A2 (ru)
CA (1) CA3052829A1 (ru)
EA (1) EA038695B1 (ru)
IL (2) IL268534B2 (ru)
MX (1) MX2019009720A (ru)
SG (1) SG11201907298WA (ru)
WO (1) WO2018152333A1 (ru)

Families Citing this family (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
SG10202107733QA (en) 2015-09-28 2021-09-29 Univ North Carolina Chapel Hill Methods and compositions for antibody-evading virus vectors
US20190085358A1 (en) * 2017-08-25 2019-03-21 Ovid Therapeutics Inc. Recombinant adeno-associated vectors
CA3075656A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
CN112272672A (zh) 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
US11987801B2 (en) 2018-05-11 2024-05-21 Massachusetts Eye And Ear Infirmary Altering tissue tropism of adeno-associated viruses
CN112703198B (zh) 2018-07-11 2025-05-30 布里格姆妇女医院 用于跨血脑屏障递送试剂的方法和组合物
CN113966399A (zh) 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
AU2019346655B2 (en) 2018-09-28 2025-03-13 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
MX2021009305A (es) 2019-02-04 2021-11-04 Freeline Therapeutics Ltd Polinucleotidos.
TW202102525A (zh) 2019-03-21 2021-01-16 美商史崔德生物公司 重組腺相關病毒載體
TW202102526A (zh) * 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
CA3137284A1 (en) * 2019-04-24 2020-10-29 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
CA3151021A1 (en) * 2019-08-14 2021-02-18 University Of Florida Research Foundation, Incorporated Aav capsid variants for gene therapy
CA3153972A1 (en) * 2019-09-09 2021-03-18 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
US11905523B2 (en) 2019-10-17 2024-02-20 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C
EP4051324A4 (en) * 2019-10-28 2023-11-29 University Of Florida Research Foundation, Incorporated Gene therapy vectors
KR20220116459A (ko) * 2019-11-22 2022-08-23 더 칠드런스 호스피탈 오브 필라델피아 아데노(adeno)-관련 바이러스 벡터 변이체
AU2021206256A1 (en) * 2020-01-10 2022-07-28 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
CA3171166A1 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
BR112022016965A2 (pt) * 2020-02-25 2022-12-06 Childrens Medical Res Institute Polipeptídeos e vetores de capsídeo do vírus adenoassociado
EP4125977A4 (en) * 2020-05-05 2024-09-25 Duke University CROSS-SPECIES COMPATIBLE ADENO-ASSOCIATED VIRUS COMPOSITIONS AND METHODS OF USE THEREOF
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
WO2022076750A2 (en) * 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
US20240060087A1 (en) * 2021-01-08 2024-02-22 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
US20240082351A1 (en) * 2021-01-13 2024-03-14 Duke University Compositions for and methods of improving fluid flux in the brain
JP2024523707A (ja) 2021-07-08 2024-06-28 テナヤ セラピューティクス， インコーポレイテッド 遺伝子治療のための最適化された発現カセット
WO2023288184A2 (en) * 2021-07-14 2023-01-19 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for treating chronic pain and for retrograde transduction of neurons
CN113480615B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
CN113563430B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CA3229580A1 (en) * 2021-08-20 2023-02-23 Anna Nikolaevna STRELKOVA Method for producing modified adeno-associated virus capsid
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
EP4392435A1 (en) * 2021-08-25 2024-07-03 Children's Medical Research Institute Modified aav capsid polypeptides and vectors
JP2024532891A (ja) * 2021-08-25 2024-09-10 チルドレンズ・メディカル・リサーチ・インスティテュート 改変ａａｖカプシドおよびベクター
EP4413019A2 (en) * 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for cns tropic delivery
CN114516901B (zh) * 2022-03-11 2023-01-17 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
WO2023201277A1 (en) * 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
TW202421787A (zh) 2022-09-06 2024-06-01 美商特納亞治療股份有限公司 保護心臟之心臟病療法
KR20240048599A (ko) * 2022-10-06 2024-04-16 주식회사 글루진테라퓨틱스 폐 기관지 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2025015388A1 (en) * 2023-07-20 2025-01-23 Children's Medical Research Institute Modified cardiotropic aav capsid polypeptides and vectors
WO2025121957A1 (ko) * 2023-12-06 2025-06-12 주식회사 글루진테라퓨틱스 뇌 성상교세포 특이적 유전자 전달이 가능한 아데노부속바이러스 변이체와 이의 응용

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE454445T1 (de) * 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
DE19933288A1 (de) * 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
EP1496944B1 (en) * 2002-05-01 2008-08-20 University of Florida Research Foundation, Inc. Improved raav expression systems for genetic modification of specific capsid proteins
US8889641B2 (en) * 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
EP2782596A4 (en) * 2011-11-22 2015-07-29 Philadelphia Children Hospital VIRUS VECTORS FOR HIGHLY EFFICIENT TRANSGENIC ADMINISTRATION
PL2900686T3 (pl) * 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2017015102A1 (en) * 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells

Also Published As

Publication number Publication date
JP2023052541A (ja) 2023-04-11
EP3583220A1 (en) 2019-12-25
JP7574508B2 (ja) 2024-10-29
IL268534B1 (en) 2024-06-01
SG11201907298WA (en) 2019-09-27
CN110520536A (zh) 2019-11-29
WO2018152333A1 (en) 2018-08-23
MX2019009720A (es) 2019-10-07
BR112019016769A2 (pt) 2020-05-26
AU2018221738B2 (en) 2024-06-27
US20240209031A1 (en) 2024-06-27
CN110520536B (zh) 2024-07-09
JP2020505936A (ja) 2020-02-27
JP7291397B2 (ja) 2023-06-15
KR20190117571A (ko) 2019-10-16
CN118978574A (zh) 2024-11-19
WO2018152333A9 (en) 2019-01-24
IL268534B2 (en) 2024-10-01
CA3052829A1 (en) 2018-08-23
IL312788A (en) 2024-07-01
US11905312B2 (en) 2024-02-20
US20190367562A1 (en) 2019-12-05
EP3583220A4 (en) 2021-07-07
EA038695B1 (ru) 2021-10-06
AU2018221738A1 (en) 2019-08-22
IL268534A (en) 2019-09-26

Similar Documents

Publication Publication Date Title
EA201991911A1 (ru) Способы и композиции для переноса генов по сосудистой сети
EA202092362A1 (ru) Вирусные векторы для нацеливания на ткани глаза
MX2020010466A (es) Vectores de virus que evitan anticuerpos.
WO2017058892A3 (en) Methods and compositions for antibody-evading virus vectors
PH12020550094A1 (en) Adeno-associated virus variant capsids and methods of use thereof
EA202192501A1 (ru) Рекомбинантные аденоассоциированные вирусные векторы
MX2020010464A (es) Vectores de virus que evitan anticuerpos.
BR112019002904A2 (pt) métodos e composições para transferência gênica direcionada
PH12018502387A1 (en) Adeno-associated virus variant capsids and methods of use thereof
PH12020550706A1 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
MX2022015079A (es) Variantes de hsd17b13 y usos de las mismas.
ZA201705628B (en) Methods and compositions for treating genetic eye diseases
MX388331B (es) Variantes de virus adenoasociado y métodos de uso de estas
EA201892561A1 (ru) Гибридные белки gdf15 и их применение
WO2015158749A3 (de) Viraler vektor für den zielgerichteten gentransfer in gehirn und rückenmark
PH12021552638A1 (en) Compositions useful for treatment of pompe disease
EA202091352A1 (ru) Генотерапия мукополисахаридоза iiib
EA202090628A1 (ru) Система экспрессии paramyxoviridae
MX390966B (es) Vacunas contra el dengue
EA201890187A1 (ru) Рекомбинантные вирусоподобные частицы (vlp) с использованием протеина группового антигена (gag) вируса бычьего иммунодефицита
EA201992558A1 (ru) Варианты белка bmp7 человека
EA202092353A1 (ru) Уклоняющиеся от антител вирусные векторы
EA202092363A1 (ru) Уклоняющиеся от антител вирусные векторы
EA201990955A1 (ru) Разработка капсидов aav
EA202192973A1 (ru) Композиции, полезные для лечения болезни помпе