MX353930B - Composiciones y metodos para alterar la especificidad tisular y mejorar la transferencia de genes mediada por vector viral adeno-asociado 9. - Google Patents

Composiciones y metodos para alterar la especificidad tisular y mejorar la transferencia de genes mediada por vector viral adeno-asociado 9.

Info

Publication number
MX353930B
MX353930B MX2013009538A MX2013009538A MX353930B MX 353930 B MX353930 B MX 353930B MX 2013009538 A MX2013009538 A MX 2013009538A MX 2013009538 A MX2013009538 A MX 2013009538A MX 353930 B MX353930 B MX 353930B
Authority
MX
Mexico
Prior art keywords
aav9
galactose
vector
cells
cell surface
Prior art date
Application number
MX2013009538A
Other languages
English (en)
Other versions
MX2013009538A (es
Inventor
James M Wilson
Luc H Vandenberghe
Christie L Bell
Original Assignee
Univ Pennsylvania
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 Pennsylvania filed Critical Univ Pennsylvania
Publication of MX2013009538A publication Critical patent/MX2013009538A/es
Publication of MX353930B publication Critical patent/MX353930B/es

Links

Classifications

    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/66—Phosphorus compounds
    • A61K31/683—Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols
    • A61K31/685—Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols one of the hydroxy compounds having nitrogen atoms, e.g. phosphatidylserine, lecithin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral 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
    • 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
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043—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
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14045—Special targeting system for 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

Abstract

La presente invención se refiere a una composición , caracterizada porque comprende un vector viral del virus adeno-asociado (AAV) de clado F que tiene un dominio de unión del receptor de superficie celular de ßgalactosa adecuado para su uso en una combinación con una neurominidasa que proporciona medios para la escisión enzimática de la ß-galactosa o una lectina que se une a la ß-galactosa, siendo dicha combinación adecuada para su uso en un sujeto conocido por tener células con un glicano de superficie celular que tiene ácido siálico terminal y una penúltima ß-galactosa, y en donde dicho vector AAV además comprende un minigen que tiene repeticiones terminales invertidas de AAV y un gen heterólogo operativamente enlazado a secuencias reguladoras que dirigen su expresión en una célula huésped un vector de AAV9 recombinante.
MX2013009538A 2011-02-17 2012-02-17 Composiciones y metodos para alterar la especificidad tisular y mejorar la transferencia de genes mediada por vector viral adeno-asociado 9. MX353930B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161443879P 2011-02-17 2011-02-17
PCT/US2012/025550 WO2012112832A1 (en) 2011-02-17 2012-02-17 Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer

Publications (2)

Publication Number Publication Date
MX2013009538A MX2013009538A (es) 2013-09-26
MX353930B true MX353930B (es) 2018-02-02

Family

ID=45757234

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2013009538A MX353930B (es) 2011-02-17 2012-02-17 Composiciones y metodos para alterar la especificidad tisular y mejorar la transferencia de genes mediada por vector viral adeno-asociado 9.

Country Status (16)

Country Link
US (5) US9884071B2 (es)
EP (1) EP2675902B1 (es)
JP (1) JP6224459B2 (es)
KR (1) KR101956885B1 (es)
CN (1) CN103502458B (es)
AU (1) AU2012219380B2 (es)
BR (1) BR112013020734A2 (es)
CA (1) CA2826316C (es)
DK (1) DK2675902T3 (es)
ES (1) ES2724800T3 (es)
HU (1) HUE043921T2 (es)
MX (1) MX353930B (es)
PL (1) PL2675902T3 (es)
SG (3) SG192268A1 (es)
TR (1) TR201908020T4 (es)
WO (1) WO2012112832A1 (es)

Families Citing this family (101)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2874298T3 (es) * 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP3567106A1 (en) 2010-04-23 2019-11-13 University of Massachusetts Cns targeting aav vectors and methods of use thereof
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
EP4234571A3 (en) * 2011-02-10 2023-09-27 The University of North Carolina at Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
EP2675902B1 (en) 2011-02-17 2019-03-27 The Trustees Of The University Of Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
EP2699680B1 (en) 2011-04-21 2017-12-27 University of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
US9677088B2 (en) 2012-05-09 2017-06-13 Oregon Health & Science University Adeno associated virus plasmids and vectors
EP2749569A1 (en) 2012-12-28 2014-07-02 Annibale Alessandro Puca Variant of BPIFB4 protein
US20140219964A1 (en) 2013-02-07 2014-08-07 Children's Medical Center Corporation Methods for inducing cardiomyocyte proliferation
WO2014160092A1 (en) * 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
JP6396988B2 (ja) * 2013-03-15 2018-09-26 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 二重グリカン結合性ａａｖベクターのための方法および組成物
CA2901328C (en) 2013-03-15 2023-08-01 The Trustees Of The University Of Pennsylvania Compositions and methods for treating mps1
KR20160127832A (ko) 2014-03-21 2016-11-04 젠자임 코포레이션 색소성 망막염의 유전자 치료
WO2015164757A1 (en) * 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CA2961523A1 (en) 2014-09-16 2016-03-24 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
EP4012035A1 (en) 2014-09-16 2022-06-15 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
EP3200830B1 (en) 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
US10480011B2 (en) 2014-10-21 2019-11-19 University Of Massachusetts Recombinant AAV variants and uses thereof
CA2966620A1 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
EP3218484A4 (en) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US10570395B2 (en) 2014-11-14 2020-02-25 Voyager Therapeutics, Inc. Modulatory polynucleotides
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
TWI745285B (zh) 2015-01-20 2021-11-11 美商健臻公司 用於重組病毒粒子特徵化之分析性超速離心法
JP2018506304A (ja) 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ
MX2017010370A (es) 2015-02-10 2017-12-14 Genzyme Corp Suministro mejorado de particulas virales al cuerpo estriado y al cortex.
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
SG10201910750WA (en) 2015-05-16 2020-01-30 Genzyme Corp Gene editing of deep intronic mutations
CA2988471A1 (en) * 2015-07-28 2017-02-02 University Of Massachusetts Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US11253576B2 (en) 2015-10-22 2022-02-22 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
WO2017100704A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
WO2017106313A1 (en) 2015-12-15 2017-06-22 Genzyme Corporation Adeno-associated viral vectors for treating mucolipidosis type ii
EP3411059A4 (en) 2016-02-02 2019-10-16 University Of Massachusetts METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
KR20180121899A (ko) 2016-02-03 2018-11-09 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 I형 점액다당류증을 치료하기 위한 유전자 요법
EP3413928B1 (en) 2016-02-12 2022-04-20 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
EP3440210A4 (en) 2016-04-05 2019-11-27 University of Massachusetts COMPOSITIONS AND METHODS FOR SELECTIVE INHIBITION OF EXPRESSION OF GRAINHEAD PROTEIN
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
KR20230125339A (ko) 2016-04-15 2023-08-29 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
UA124343C2 (uk) 2016-05-13 2021-09-01 4Д Молекьюлар Терапьютікс Інк. Капсиди аденоасоційованого вірусу і спосіб його використання
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CN106093369B (zh) * 2016-06-29 2017-10-20 福建农林大学 Bt水稻对非靶标害虫褐飞虱的安全性评价方法
KR20230078834A (ko) 2016-08-15 2023-06-02 젠자임 코포레이션 Aav의 검출 방법
CA3035522A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
EP3526333A4 (en) 2016-10-13 2020-07-29 University of Massachusetts AAV CAPSIDE DESIGNS
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
UA127455C2 (uk) 2017-09-20 2023-08-30 4Д Молекьюлар Терапьютикс Інк. Капсид аденоасоційованого вірусу і спосіб його застосування
JP7449223B2 (ja) 2017-09-22 2024-03-13 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 ムコ多糖症ｉｉ型を治療するための遺伝子療法
JP7248658B2 (ja) 2017-09-22 2023-03-29 ジェンザイム・コーポレーション バリアントＲＮＡｉ
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP3697905A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
ES2946747T3 (es) 2017-11-27 2023-07-25 4D Molecular Therapeutics Inc Variantes de cápsides de virus adenoasociado y su utilización para inhibir la angiogénesis
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3746113A4 (en) * 2018-02-01 2022-03-09 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR PAH GENE TRANSFER AND METHODS OF USE
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
BR112020020223A2 (pt) 2018-04-05 2021-01-19 Genethon Sorotipo de vírus adeno-associado híbrido recombinante entre aav9 e aavrh74 com tropismo pelo fígado reduzido
JP2021523702A (ja) * 2018-05-04 2021-09-09 オレゴン ヘルス アンド サイエンス ユニバーシティ ヒト抗ａａｖ２カプシドポリクローナル抗体エピトープ
EP3807404A1 (en) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
CA3107462A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
SG11202101072UA (en) 2018-08-03 2021-03-30 Genzyme Corp Variant rnai against alpha-synuclein
EP3861113A1 (en) 2018-10-04 2021-08-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
WO2020072844A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
KR20210076051A (ko) 2018-10-12 2021-06-23 젠자임 코포레이션 간-특이적 유전자 대체 요법에 의한 중증 pku 치료를 위한 개선된 인간 pah의 생성
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
CA3116701A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. Expression vectors for large-scale production of raav in the baculovirus/sf9 system
CN113924115A (zh) * 2019-01-31 2022-01-11 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法
EP3931335A2 (en) 2019-02-25 2022-01-05 Novartis AG Compositions and methods to treat bietti crystalline dystrophy
CA3130731A1 (en) 2019-02-25 2020-09-03 Friedrich Miescher Institute For Biomedical Research Compositions and methods to treat bietti crystalline dystrophy
US20220047618A1 (en) 2019-02-28 2022-02-17 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
EP3953484A4 (en) * 2019-04-12 2023-04-12 University of Massachusetts AAV-MEDIATED GENE THERAPY FOR LEUCINOSIS
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
US10801042B1 (en) 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
WO2021087361A1 (en) * 2019-11-01 2021-05-06 Sangamo Therapeutics, Inc. Zinc finger nuclease variants for treating or preventing lysosomal storage diseases
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
BR112023003929A2 (pt) 2020-10-01 2023-04-11 Genzyme Corp Cassete de expressão de pah humano para tratamento de pku por terapia de reposição gênica direcionada ao fígado
BR112023015303A2 (pt) 2021-02-01 2023-11-14 Regenxbio Inc Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito
CA3227103A1 (en) 2021-07-30 2023-02-02 Matthew P. GEMBERLING Compositions and methods for modulating expression of frataxin (fxn)
CA3227105A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
WO2023147304A1 (en) * 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023214346A1 (en) 2022-05-06 2023-11-09 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2024015881A2 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation

Family Cites Families (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5774090A (en) 1980-10-27 1982-05-10 Shionogi & Co Ltd Novel beta-galactosidase inhibitor, gt-2558, its derivative, and production
US4886876A (en) 1983-03-31 1989-12-12 Scripps Clinic And Research Foundation Factor VIII coagulant polypeptides
US4542104A (en) 1983-04-06 1985-09-17 The Board Of Trustees Of The Leland Stanford Jr. Univ. Phycobiliprotein fluorescent conjugates
US4757006A (en) 1983-10-28 1988-07-12 Genetics Institute, Inc. Human factor VIII:C gene and recombinant methods for production
JPH07106156B2 (ja) 1983-10-28 1995-11-15 ジェネティックス、インスティチュ−ト ファクタ−▲ｖｉｉｉ▼および関連生産物の製造
US5045455A (en) 1984-01-12 1991-09-03 Chiron Corporation Factor VIII:C cDNA cloning and expression
IL74909A (en) 1984-04-20 1992-01-15 Genentech Inc Preparation of functional human factor viii and dna sequences,expression vectors,transformed microorganisms and cell lines used therein
US4965199A (en) 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
EP0182448A3 (en) 1984-08-24 1987-10-28 Genetics Institute, Inc. Production of factor viii and related products
FI98829C (fi) 1986-01-27 1997-08-25 Chiron Corp Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US5451521A (en) 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
US5149637A (en) 1987-04-06 1992-09-22 Scripps Clinic & Research Foundation Recombinant Factor VIIIC fragments
IL86693A (en) 1987-06-12 1994-06-24 Stichting Centraal Lab Proteins that have the activity IIIV of the blood, a process for their preparation that uses cells are produced through genetic engineering and pharmaceutical preparations that contain them
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
US5004803A (en) 1988-11-14 1991-04-02 Genetics Institute, Inc. Production of procoagulant proteins
ZA909167B (en) 1989-11-17 1993-10-15 Chiron Corp Protein complexes having Factor VIII:C activity and production thereof
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
US5272257A (en) 1990-05-21 1993-12-21 Coulter Corporation Method of preferential labelling of a phycobiliprotein with an amine-reactive dye for use in a multiple color assay and product for such use
US5171846A (en) 1990-05-21 1992-12-15 Coulter Corporation Method of preferential labelling of a phycobiliprotein with a second dye for use in a multiple color assay and product for such use
US5240640A (en) 1990-06-04 1993-08-31 Coulter Corporation In situ use of gelatin or an aminodextran in the preparation of uniform ferrite particles
US5466609A (en) 1990-10-31 1995-11-14 Coulter Corporation Biodegradable gelatin-aminodextran particle coatings of and processes for making same
US5639620A (en) 1990-10-31 1997-06-17 Coulter Corporation Polymeric particles having a biodegradable gelatin or aminodextran coating and process for making same
SE468050C (sv) 1991-03-15 1998-02-11 Pharmacia & Upjohn Ab Rekombinant derivat av human faktor VIII
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
US5248771A (en) 1991-06-26 1993-09-28 Triumf Process for preparing no-carrier-added radiohalogenated vinyl nucleosides
CA2078721A1 (en) 1991-09-24 1993-03-25 Hiroshi Yonemura Process for preparing human coagulation factor viii protein complex
US5248772A (en) 1992-01-29 1993-09-28 Coulter Corporation Formation of colloidal metal dispersions using aminodextrans as reductants and protective agents
AU5602394A (en) 1992-11-13 1994-06-08 Duke University Chimeric blood coagulation proteins
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
ATE272122T1 (de) 1993-06-10 2004-08-15 Bayer Ag Vektoren und zelllinien von säugetieren mit erhöhter produktivität
CA2164646A1 (en) 1993-06-11 1994-12-22 Wade E. Bolton Anti-cd3 antibody-aminodextran conjugates for induction of t-cell activation and proliferation
US5681746A (en) 1994-12-30 1997-10-28 Chiron Viagene, Inc. Retroviral delivery of full length factor VIII
AU6486196A (en) 1995-07-11 1997-02-10 Chiron Corporation Novel factor viii:c polypeptide analogs with altered protease sites
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
US6096273A (en) 1996-11-05 2000-08-01 Clinical Micro Sensors Electrodes linked via conductive oligomers to nucleic acids
US5945293A (en) 1997-10-09 1999-08-31 Coulter International Corp. Protein-colloidal metal-aminodextran coated particle and methods of preparation and use
US6074884A (en) 1997-10-09 2000-06-13 Coulter International Corp. Stable protein-nickel particles and methods of production and use thereof
US6117643A (en) 1997-11-25 2000-09-12 Ut Battelle, Llc Bioluminescent bioreporter integrated circuit
US6197503B1 (en) 1997-11-26 2001-03-06 Ut-Battelle, Llc Integrated circuit biochip microsystem containing lens
US6287857B1 (en) * 1998-02-09 2001-09-11 Genzyme Corporation Nucleic acid delivery vehicles
JP4028964B2 (ja) 1998-09-09 2008-01-09 日立ソフトウエアエンジニアリング株式会社 バイオチップ及びバイオチップの使用方法
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
ES2874298T3 (es) * 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
WO2006119432A2 (en) * 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
US10920244B2 (en) 2009-04-30 2021-02-16 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
JP5458245B2 (ja) 2010-06-25 2014-04-02 国立大学法人九州工業大学 動作制御装置、その方法、及びプログラム
WO2012109214A1 (en) 2011-02-07 2012-08-16 The University Of North Carolina At Chapel Hill Targeted transduction of aav vectors
EP4234571A3 (en) 2011-02-10 2023-09-27 The University of North Carolina at Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
EP2675902B1 (en) 2011-02-17 2019-03-27 The Trustees Of The University Of Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer

Also Published As

Publication number Publication date
JP6224459B2 (ja) 2017-11-01
CA2826316C (en) 2021-04-27
US11766448B2 (en) 2023-09-26
BR112013020734A2 (pt) 2017-06-13
JP2014507154A (ja) 2014-03-27
CA2826316A1 (en) 2012-08-23
TR201908020T4 (tr) 2019-06-21
US10406173B2 (en) 2019-09-10
MX2013009538A (es) 2013-09-26
ES2724800T3 (es) 2019-09-16
US20210145857A1 (en) 2021-05-20
CN103502458B (zh) 2016-11-16
US9884071B2 (en) 2018-02-06
EP2675902B1 (en) 2019-03-27
US20240082288A1 (en) 2024-03-14
US20180185405A1 (en) 2018-07-05
EP2675902A1 (en) 2013-12-25
CN103502458A (zh) 2014-01-08
AU2012219380A1 (en) 2013-08-29
KR20140046407A (ko) 2014-04-18
PL2675902T3 (pl) 2019-08-30
US20200101099A1 (en) 2020-04-02
AU2012219380B2 (en) 2017-04-13
US10918658B2 (en) 2021-02-16
SG10201601110VA (en) 2016-03-30
US20130323226A1 (en) 2013-12-05
HUE043921T2 (hu) 2019-09-30
SG10202007803QA (en) 2020-09-29
WO2012112832A1 (en) 2012-08-23
DK2675902T3 (da) 2019-06-03
KR101956885B1 (ko) 2019-03-12
SG192268A1 (en) 2013-08-30

Similar Documents

Publication Publication Date Title
MX353930B (es) Composiciones y metodos para alterar la especificidad tisular y mejorar la transferencia de genes mediada por vector viral adeno-asociado 9.
PH12018501168A1 (en) Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
RU2022103603A (ru) Направляемые нуклеиновыми кислотами нуклеазы
MX2021014700A (es) Variantes de vector del virus asociado a adeno para edicion genomica de alta eficacia y sus metodos.
MX2018011791A (es) Compuestos para transduccion viral mejorada.
JP2020518276A5 (es)
JP2014507154A5 (es)
SG195181A1 (en) Simian adenovirus and hybrid adenoviral vectors
TR201906398T4 (tr) Salgı bezleriyle ilişkili virüs yapılarını içeren iletken hava yolu hücrelerinin hedeflenmesine yönelik bileşimler.
NZ586238A (en) Methods and compositions for immunizing pigs against porcine circovirus
JP2017536116A5 (es)
BR112012025364A2 (pt) método para geração de partícula semelhante a vírus de parvovírus b19
NZ701693A (en) Composition and methods for highly efficient gene transfer using aav capsid variants
NZ600733A (en) Engineered landing pads for gene targeting in plants
WO2006094976A3 (en) Expression enhancing intron sequences
JP2009509556A5 (es)
BR112014012162A2 (pt) partícula similar a vírus, construto de ácidos nucleicos, vetor, célula hospedeira, vegetal ou tecido vegetal, semente ou progênie do vegetal ou tecido vegetal, composição, métodos para isolar e purificar molécula carregada alvo e para proteger molécula alvo contra hidrólise e processo para purificar partículas similares e vírus
AU2019268128A1 (en) Viral vector nanocapsule for targeting gene therapy and its preparation
WO2014026110A3 (en) Compounds for improved viral transduction
WO2013030176A3 (en) Live attenuated influenza virus
WO2012075462A3 (en) Methods and compositions for treatment of muscular dystrophy
WO2008133137A1 (ja) 遺伝子治療のためのベクター
DE602006016756D1 (de) Auf cis einwirkende regulierende elemente aus tripsacum-dactyloiden
JP2013531967A5 (es)
WO2008058752A3 (en) Lentiviral vectors for gene transfer in quiescent (g0) cells

Legal Events

Date Code Title Description
HH Correction or change in general
FG Grant or registration