ATE369434T1 - Adenoassoziierte virusvektoren und ihre verwendungen - Google Patents

Adenoassoziierte virusvektoren und ihre verwendungen

Info

Publication number
ATE369434T1
ATE369434T1 AT99924404T AT99924404T ATE369434T1 AT E369434 T1 ATE369434 T1 AT E369434T1 AT 99924404 T AT99924404 T AT 99924404T AT 99924404 T AT99924404 T AT 99924404T AT E369434 T1 ATE369434 T1 AT E369434T1
Authority
AT
Austria
Prior art keywords
adeno
associated virus
virus vectors
provides
isolated
Prior art date
Application number
AT99924404T
Other languages
English (en)
Inventor
John Engelhardt
Dongsheng Duan
Original Assignee
Univ Iowa Res Found
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 Univ Iowa Res Found filed Critical Univ Iowa Res Found
Application granted granted Critical
Publication of ATE369434T1 publication Critical patent/ATE369434T1/de

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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
    • 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • 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
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • 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
    • C12N2750/14141—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)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Toxicology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pulmonology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
AT99924404T 1998-05-20 1999-05-20 Adenoassoziierte virusvektoren und ihre verwendungen ATE369434T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US8616698P 1998-05-20 1998-05-20
US09/276,625 US6436392B1 (en) 1998-05-20 1999-03-25 Adeno-associated virus vectors

Publications (1)

Publication Number Publication Date
ATE369434T1 true ATE369434T1 (de) 2007-08-15

Family

ID=26774429

Family Applications (1)

Application Number Title Priority Date Filing Date
AT99924404T ATE369434T1 (de) 1998-05-20 1999-05-20 Adenoassoziierte virusvektoren und ihre verwendungen

Country Status (10)

Country Link
US (4) US6436392B1 (de)
EP (1) EP1082444B1 (de)
JP (1) JP2002515257A (de)
AT (1) ATE369434T1 (de)
AU (1) AU759093B2 (de)
CA (1) CA2328447C (de)
DE (1) DE69936778T2 (de)
ES (1) ES2291026T3 (de)
PT (1) PT1082444E (de)
WO (1) WO1999060146A1 (de)

Families Citing this family (129)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6924128B2 (en) * 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US6995006B2 (en) * 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US6436392B1 (en) * 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
US6893865B1 (en) * 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
WO2003104467A1 (en) 2002-04-25 2003-12-18 Crucell Holland B.V. Means and methods for the production of adenovirus vectors
JP4969002B2 (ja) * 1999-06-08 2012-07-04 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン ｒＡＡＶ形質導入を増加するための化合物および方法
US7122335B1 (en) 1999-06-08 2006-10-17 University Of Iowa Research Foundation Compounds and methods to enhance rAAV transduction
US7241447B1 (en) * 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
EP1224313A1 (de) * 1999-10-07 2002-07-24 University of Iowa Research Foundation Adeno-assoziierte viren und ihre verwendungen
AU784420B2 (en) * 1999-10-07 2006-03-30 University Of Iowa Research Foundation, The Adeno-associated viruses and uses thereof
JP2003511082A (ja) * 1999-10-12 2003-03-25 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 第ｖｉｉｉ因子をコードするアデノ随伴ウィルスベクターとその使用法
US6544786B1 (en) * 1999-10-15 2003-04-08 University Of Pittsburgh Of The Commonwealth Of Higher Education Method and vector for producing and transferring trans-spliced peptides
WO2001066782A1 (en) * 2000-03-03 2001-09-13 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral compositions and methods
US8067156B2 (en) * 2001-05-31 2011-11-29 The Rockefeller University Method for generating replication defective viral vectors that are helper free
US8241622B2 (en) * 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
US7829694B2 (en) * 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7618948B2 (en) * 2002-11-26 2009-11-17 Medtronic, Inc. Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA
US7994149B2 (en) 2003-02-03 2011-08-09 Medtronic, Inc. Method for treatment of Huntington's disease through intracranial delivery of sirna
US7732591B2 (en) * 2003-11-25 2010-06-08 Medtronic, Inc. Compositions, devices and methods for treatment of huntington's disease through intracranial delivery of sirna
WO2004089423A2 (en) * 2003-03-31 2004-10-21 University Of Iowa Research Foundation Pharmico-gene theraphy of epithelial sodium channel associated disoders
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
EP1773401B1 (de) * 2004-06-21 2013-01-02 Medtronic, Inc. Medizinische systeme und verfahren zur abgabe von zusammensetzungen an zellen
US20060257912A1 (en) * 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
US7902352B2 (en) * 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
ATE522626T1 (de) * 2005-06-28 2011-09-15 Medtronic Inc Verfahren und nukleotid-sequenzen, die bevorzugt die expression eines mutierten huntingtin-genes unterdrücken
US20080280843A1 (en) * 2006-05-24 2008-11-13 Van Bilsen Paul Methods and kits for linking polymorphic sequences to expanded repeat mutations
US9133517B2 (en) * 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
EP2019964A2 (de) * 2006-04-28 2009-02-04 University of Iowa Research Foundation Verfahren und verbindungen zur modulation der parvovirusübertragung von säugerzellen oder zur änderung von virusinfektionen verfahren zur identifizierung eines viralen rezeptors oder co-rezeptors
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US20080039415A1 (en) * 2006-08-11 2008-02-14 Gregory Robert Stewart Retrograde transport of sirna and therapeutic uses to treat neurologic disorders
US8324367B2 (en) 2006-11-03 2012-12-04 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
US9375440B2 (en) * 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
US7988668B2 (en) * 2006-11-21 2011-08-02 Medtronic, Inc. Microsyringe for pre-packaged delivery of pharmaceuticals
US7819842B2 (en) * 2006-11-21 2010-10-26 Medtronic, Inc. Chronically implantable guide tube for repeated intermittent delivery of materials or fluids to targeted tissue sites
US20080171906A1 (en) * 2007-01-16 2008-07-17 Everaerts Frank J L Tissue performance via hydrolysis and cross-linking
WO2011097456A2 (en) 2010-02-05 2011-08-11 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
CN107320734A (zh) 2011-04-29 2017-11-07 西莱克塔生物科技公司 从合成纳米载体中控制释放免疫抑制剂
EP2764119A2 (de) 2011-10-05 2014-08-13 Genelux Corporation Methoden zum nachweis der replikation oder kolonisierung eines biologischen therapiemittels
WO2013138522A2 (en) 2012-03-16 2013-09-19 Genelux Corporation Methods for assessing effectiveness and monitoring oncolytic virus treatment
WO2013158265A1 (en) 2012-04-20 2013-10-24 Genelux Corporation Imaging methods for oncolytic virus therapy
WO2014055960A1 (en) 2012-10-05 2014-04-10 Genelux Corporation Energy absorbing-based diagnostic and therapeutic methods employing nucleic acid molecules encoding chromophore-producing enzymes
AU2014251099B2 (en) 2013-04-08 2019-01-17 The University Of Kansas Chimeric adeno-associated virus/ bocavirus parvovirus vector
CN105307641A (zh) 2013-05-03 2016-02-03 西莱克塔生物科技公司 用于降低不期望体液免疫应答的给药组合
WO2015103438A2 (en) 2014-01-02 2015-07-09 Genelux Corporation Oncolytic virus adjunct therapy with agents that increase virus infectivity
CN106460062A (zh) 2014-05-05 2017-02-22 美敦力公司 用于scd、crt、crt‑d或sca治疗识别和/或选择的方法和组合物
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EA201790551A1 (ru) 2014-09-07 2017-07-31 Селекта Байосайенсиз, Инк. Способы и композиции для ослабления иммунных ответов к пропускающему экзон вирусному трансферному вектору
AU2015332533B2 (en) 2014-10-14 2018-08-30 Halozyme, Inc. Compositions of adenosine deaminase-2 (ADA2), variants thereof and methods of using same
EP3215191B1 (de) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc-polynukleotide zur behandlung von morbus parkinson
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
WO2017155973A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
SG11201808457PA (en) 2016-04-15 2018-10-30 Alpine Immune Sciences Inc Icos ligand variant immunomodulatory proteins and uses thereof
AU2017248830B2 (en) 2016-04-15 2023-03-09 Alpine Immune Sciences, Inc. CD80 variant immunomodulatory proteins and uses thereof
EP3448874A4 (de) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Zusammensetzungen zur behandlung einer erkrankung
EP3448987A4 (de) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. Zusammensetzungen zur behandlung einer erkrankung
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
US11471488B2 (en) 2016-07-28 2022-10-18 Alpine Immune Sciences, Inc. CD155 variant immunomodulatory proteins and uses thereof
WO2018022945A1 (en) 2016-07-28 2018-02-01 Alpine Immune Sciences, Inc. Cd112 variant immunomodulatory proteins and uses thereof
EP3491013A1 (de) 2016-07-28 2019-06-05 Alpine Immune Sciences, Inc. Cd155-variante immunmodulatorische proteine und verwendungen davon
WO2018039375A1 (en) 2016-08-23 2018-03-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
EP3831281B1 (de) 2016-08-30 2026-02-25 The Regents of The University of California Vorrichtungen und systeme für biomedizinische zielführung und verabreichung
EP3872180A1 (de) 2016-10-20 2021-09-01 Alpine Immune Sciences, Inc. Sekretierbare variante immunmodulatorische proteine und manipulierte zelltherapie
EP3535293A1 (de) 2016-11-07 2019-09-11 Crossbeta Biosciences B.V. Neuartiges amyloid-beta-oligomerspezifisches bindungsmolekül
JP2020506890A (ja) 2017-01-07 2020-03-05 セレクタ バイオサイエンシーズ インコーポレーテッドＳｅｌｅｃｔａ Ｂｉｏｓｃｉｅｎｃｅｓ，Ｉｎｃ． 合成ナノキャリアにカップリングした免疫抑制剤のパターン化された投与
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
AU2018219895C1 (en) 2017-02-09 2024-10-03 Indapta Therapeutics, Inc. Engineered Natural Killer (NK) cells and compositions and methods thereof
EP4599848A3 (de) 2017-03-11 2025-12-03 Cartesian Therapeutics, Inc. Verfahren und zusammensetzungen in zusammenhang mit einer kombinierten behandlung mit entzündungshemmenden mitteln und synthetischen nanoträgern mit einem immunsuppressivum
MX2019010887A (es) 2017-03-16 2019-10-15 Alpine Immune Sciences Inc Proteinas inmunomoduladoras de cb80 variante y usos de estas.
JP2020511144A (ja) 2017-03-16 2020-04-16 アルパイン イミューン サイエンシズ インコーポレイテッド Ｐｄ−ｌ２バリアント免疫調節タンパク質及びその使用
FI3596116T3 (fi) 2017-03-16 2023-11-09 Alpine Immune Sciences Inc Immunomodulatorisia pd-l1-varianttiproteiineja ja niiden käyttöjä
CN110506060B (zh) 2017-03-30 2024-05-07 昆士兰大学 嵌合分子及其用途
EP3618839A4 (de) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von amyotropher lateralsklerose (als)
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
US11572544B2 (en) 2017-06-14 2023-02-07 The Children's Medical Center Corporation Hematopoietic stem and progenitor cells derived from hemogenic endothelial cells by episomal plasmid gene transfer
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
WO2019040994A1 (en) 2017-09-01 2019-03-07 The Australian National University IMMUNOREGULATOR MOLECULES AND USES THEREOF
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
EP3694870A1 (de) 2017-10-10 2020-08-19 Alpine Immune Sciences, Inc. Ctla-4-variante immunmodulatorische proteine und verwendungen davon
WO2019075360A1 (en) 2017-10-13 2019-04-18 Selecta Biosciences, Inc. METHODS AND COMPOSITIONS FOR MITIGATING ANTI-VECTOR VIRAL TRANSFER IGM RESPONSES
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JP7282760B2 (ja) 2017-10-18 2023-05-29 アルパイン イミューン サイエンシズ インコーポレイテッド バリアントｉｃｏｓリガンド免疫調節タンパク質ならびに関連する組成物および方法
SG11202006148UA (en) 2018-01-03 2020-07-29 Alpine Immune Sciences Inc Multi-domain immunomodulatory proteins and methods of use thereof
IL325122A (en) 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
JP2021515037A (ja) 2018-02-26 2021-06-17 アントルクス，インコーポレーテッド 寛容原性リポソーム及びその使用方法
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN112703016B (zh) 2018-04-27 2024-11-22 分贝治疗公司 肌球蛋白15启动子及其用途
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
KR102662837B1 (ko) 2018-06-04 2024-05-03 카리디 바이오테라퓨틱스, 인크. 바이러스 치료법의 강화를 위한 세포-기반 비히클
WO2019241758A1 (en) 2018-06-15 2019-12-19 Alpine Immune Sciences, Inc. Pd-1 variant immunomodulatory proteins and uses thereof
CN112771070A (zh) 2018-07-16 2021-05-07 西莱克塔生物科技公司 Otc构建体和载体的方法和组合物
EP3823676A1 (de) 2018-07-16 2021-05-26 Selecta Biosciences, Inc. Verfahren und zusammensetzungen von mma-konstrukten und vektoren
WO2020047161A2 (en) 2018-08-28 2020-03-05 Actym Therapeutics, Inc. Engineered immunostimulatory bacterial strains and uses thereof
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN113613668A (zh) 2018-10-24 2021-11-05 百时美施贵宝公司 小型化肌营养不良蛋白及其用途
CA3116192C (en) 2018-11-06 2025-09-23 Calidi Biotherapeutics (Nevada), Inc. IMPROVED SYSTEMS FOR CELL-MEDIATED ONCOLYTIC VIRAL THERAPY
EP3884041A2 (de) 2018-11-21 2021-09-29 Indapta Therapeutics, Inc. Verfahren zur expansion des subsets von natürlichen killer (nk)-tellen und verwandte zusammensetzungen und verfahren
CN113727998A (zh) 2018-11-30 2021-11-30 高山免疫科学股份有限公司 Cd86变体免疫调节蛋白及其用途
EP3921032A4 (de) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von sensorineuralem hörverlust unter verwendung von otoferlin-doppelvektorsystemen
WO2020163761A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
KR20220004959A (ko) 2019-02-27 2022-01-12 액팀 테라퓨틱스, 인코퍼레이티드 종양, 종양-상주 면역 세포, 및 종양 미세환경을 콜로니화하기 위해 조작된 면역자극성 박테리아
ES2965341T3 (es) 2019-04-11 2024-04-12 Regenxbio Inc Métodos de cromatografía de exclusión por tamaño para la caracterización de composiciones de virus adenoasociados recombinantes
CA3138525A1 (en) 2019-04-28 2020-11-05 Selecta Biosciences, Inc. Methods for treatment of subjects with preexisting immunity to viral transfer vectors
CA3141863A1 (en) 2019-05-28 2020-12-03 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
US12553041B2 (en) 2019-06-04 2026-02-17 Cartesian Therapeutics Inc. Formulations and doses of pegylated uricase
EP3782465B1 (de) 2019-08-23 2023-08-30 Universität Potsdam Transgenes zebrafischmodell für schlaganfall
TWI896570B (zh) 2019-10-30 2025-09-11 美商迪賽博治療公司 使用耳畸蛋白雙載體系統治療感覺神經性聽力損失之組合物及方法
CA3168055A1 (en) 2020-02-21 2021-08-26 Emmanuel John Simons Compositions and methods for treating non-age-associated hearing impairment in a human subject
KR20230088306A (ko) 2020-04-22 2023-06-19 인답타 세라뷰틱스 인코포레이티드 자연 살해(ｎｋ) 세포 조성물 및 이를 생성하는 방법
US11535868B2 (en) 2020-04-29 2022-12-27 Bristol-Myers Squibb Company Miniaturized dystrophins having spectrin fusion domains and uses thereof
US12202882B2 (en) 2020-05-08 2025-01-21 Alpine Immune Sciences, Inc. APRIL and BAFF inhibitory immunomodulatory proteins and methods of use thereof
WO2022147481A1 (en) 2020-12-30 2022-07-07 Ansun Biopharma Inc. Combination therapy of an oncolytic virus delivering a foreign antigen and an engineered immune cell expressing a chimeric receptor targeting the foreign antigen
AU2022206197A1 (en) 2021-01-05 2023-07-13 Selecta Biosciences, Inc. Viral vector dosing protocols
US20230140196A1 (en) 2021-10-12 2023-05-04 Selecta Biosciences, Inc. Viral vector dosing protocols
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
WO2023086615A1 (en) 2021-11-14 2023-05-19 Selecta Biosciences, Inc. Multiple dosing with viral vectors
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
JP2025525583A (ja) 2022-07-19 2025-08-05 ランパート バイオサイエンス インコーポレイテッド 非免疫原性環状非ウイルス性ｄｎａベクター
JP2025526816A (ja) 2022-08-11 2025-08-15 カーティザン セラピューティクス，インコーポレーテッド 免疫グロブリンプロテアーゼおよびその融合体に関する組成物および方法

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5658772A (en) * 1989-12-22 1997-08-19 E. I. Du Pont De Nemours And Company Site-specific recombination of DNA in plant cells
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
AU5800894A (en) 1992-12-15 1994-07-04 Board Of Regents, The University Of Texas System Novel granulocytic precursor cell growth factor and method
US5498531A (en) 1993-09-10 1996-03-12 President And Fellows Of Harvard College Intron-mediated recombinant techniques and reagents
EP0733103B1 (de) 1993-11-09 2004-03-03 Targeted Genetics Corporation Die erzielung hoher titer des rekombinanten aav-vektors
US5750398A (en) 1993-11-30 1998-05-12 David C. Johnson Vector, element and method for inhibiting immune recognition
US5604090A (en) 1994-06-06 1997-02-18 Fred Hutchinson Cancer Research Center Method for increasing transduction of cells by adeno-associated virus vectors
US5843742A (en) * 1994-12-16 1998-12-01 Avigen Incorporated Adeno-associated derived vector systems for gene delivery and integration into target cells
EP0863913A4 (de) * 1995-08-21 2001-04-11 Univ Duke Methode zur erhölung der dichte eines antigens auf antigen-präsentierenden zellen
US5801030A (en) * 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
EP0883344B2 (de) 1995-12-15 2010-06-09 VIRxSYS Corporation Durch trans-spaltung erhaltene therapeutische molekule
EP1696036B1 (de) 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Rekombinante AAV zur Herstellung eines Medikaments für die Gentherapie von Muskelzellen
WO1998024479A1 (en) 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
US6287569B1 (en) 1997-04-10 2001-09-11 The Regents Of The University Of California Vaccines with enhanced intracellular processing
FR2763845B1 (fr) 1997-05-30 2003-04-18 Centre Nat Rech Scient Produits anti-cancereux pour le traitement de la mucoviscidose
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
EP1153612A4 (de) 1999-02-10 2003-06-18 Takashi Tsuruo Verstarker von antikrebs mitteln
US6306663B1 (en) 1999-02-12 2001-10-23 Proteinex, Inc. Controlling protein levels in eucaryotic organisms
JP4969002B2 (ja) * 1999-06-08 2012-07-04 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン ｒＡＡＶ形質導入を増加するための化合物および方法
AU784420B2 (en) 1999-10-07 2006-03-30 University Of Iowa Research Foundation, The Adeno-associated viruses and uses thereof
US7241447B1 (en) * 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
EP1224313A1 (de) 1999-10-07 2002-07-24 University of Iowa Research Foundation Adeno-assoziierte viren und ihre verwendungen
JP2003511082A (ja) 1999-10-12 2003-03-25 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 第ｖｉｉｉ因子をコードするアデノ随伴ウィルスベクターとその使用法
US6544786B1 (en) 1999-10-15 2003-04-08 University Of Pittsburgh Of The Commonwealth Of Higher Education Method and vector for producing and transferring trans-spliced peptides
WO2001066782A1 (en) 2000-03-03 2001-09-13 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral compositions and methods
JP2004514407A (ja) 2000-04-28 2004-05-20 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 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
WO2002087306A2 (en) 2001-05-01 2002-11-07 Senomyx, Inc. High throughput cell-based assay for monitoring sodium channel activity and discovery of salty taste modulating compounds
CA2453405A1 (en) 2001-07-13 2003-01-23 University Of Iowa Research Foundation Pseudotyped adeno-associated viruses and uses thereof
US8241622B2 (en) * 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
WO2004089423A2 (en) 2003-03-31 2004-10-21 University Of Iowa Research Foundation Pharmico-gene theraphy of epithelial sodium channel associated disoders

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US6436392B1 (en) 2002-08-20
CA2328447A1 (en) 1999-11-25
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US20020197237A1 (en) 2002-12-26
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AU759093B2 (en) 2003-04-03
JP2002515257A (ja) 2002-05-28
US20090265796A1 (en) 2009-10-22
PT1082444E (pt) 2007-10-29
US7803622B2 (en) 2010-09-28
DE69936778T2 (de) 2008-04-30
CA2328447C (en) 2007-04-03
DE69936778D1 (de) 2007-09-20
EP1082444B1 (de) 2007-08-08
AU4091299A (en) 1999-12-06
ES2291026T3 (es) 2008-02-16
EP1082444A1 (de) 2001-03-14

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