BR112018076394A2 - genes de minidistrofina otimizados e cassetes de expressão e seu uso - Google Patents

genes de minidistrofina otimizados e cassetes de expressão e seu uso

Info

Publication number
BR112018076394A2
BR112018076394A2 BR112018076394-2A BR112018076394A BR112018076394A2 BR 112018076394 A2 BR112018076394 A2 BR 112018076394A2 BR 112018076394 A BR112018076394 A BR 112018076394A BR 112018076394 A2 BR112018076394 A2 BR 112018076394A2
Authority
BR
Brazil
Prior art keywords
minidistrophin
genes
optimized
expression cassettes
proteins
Prior art date
Application number
BR112018076394-2A
Other languages
English (en)
Inventor
W.J. Mcphee Scott
Qiao Chunping
Li Juan
Xiao Xiao
Mcintyre Maritza
J. Samulski Richard
Original Assignee
Bamboo Therapeutics, Inc.
The University Of North Carolina At Chapel Hill
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 Bamboo Therapeutics, Inc., The University Of North Carolina At Chapel Hill filed Critical Bamboo Therapeutics, Inc.
Publication of BR112018076394A2 publication Critical patent/BR112018076394A2/pt

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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • 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
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • 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
    • 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
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Environmental Sciences (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)

Abstract

esta invenção refere-se a polinucleotídeos que codificam proteínas de minidistrofina, vetores virais compreendendo os mesmos e métodos de uso dos mesmos para liberação de minidistrofina a uma célula ou a um indivíduo.
BR112018076394-2A 2016-06-21 2017-06-20 genes de minidistrofina otimizados e cassetes de expressão e seu uso BR112018076394A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662352675P 2016-06-21 2016-06-21
US62/352,675 2016-06-21
US201762516449P 2017-06-07 2017-06-07
US62/516,449 2017-06-07
PCT/IB2017/053656 WO2017221145A1 (en) 2016-06-21 2017-06-20 Optimized mini-dystrophin genes and expression cassettes and their use

Publications (1)

Publication Number Publication Date
BR112018076394A2 true BR112018076394A2 (pt) 2019-03-26

Family

ID=59350997

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018076394-2A BR112018076394A2 (pt) 2016-06-21 2017-06-20 genes de minidistrofina otimizados e cassetes de expressão e seu uso

Country Status (19)

Country Link
US (3) US20170368198A1 (pt)
EP (1) EP3472194A1 (pt)
JP (2) JP6793758B2 (pt)
KR (1) KR102568278B1 (pt)
CN (2) CN109641944B (pt)
AU (1) AU2017281983B2 (pt)
BR (1) BR112018076394A2 (pt)
CA (1) CA2971303A1 (pt)
CO (1) CO2019000395A2 (pt)
IL (2) IL263199B2 (pt)
MX (1) MX2018015921A (pt)
MY (1) MY197846A (pt)
PE (1) PE20190563A1 (pt)
PH (1) PH12018502645A1 (pt)
RU (1) RU2753194C2 (pt)
SA (1) SA518400623B1 (pt)
SG (1) SG11201811115TA (pt)
TW (1) TWI719223B (pt)
WO (1) WO2017221145A1 (pt)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45477A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
US11534501B2 (en) 2017-10-18 2022-12-27 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
CN108660159B (zh) * 2018-04-12 2020-10-30 四川大学 重组蝙蝠腺相关病毒载体及其用途
BR112020021179A2 (pt) * 2018-04-16 2021-03-02 The Trustees Of The University Of Pennsylvania composições e métodos para tratar distrofia muscular de duchenne
AU2019368307A1 (en) * 2018-10-24 2021-06-10 Bristol-Myers Squibb Company Miniaturized dystrophins and uses thereof
JP2022523038A (ja) * 2019-02-02 2022-04-21 ユニバーシティ オブ ピッツバーグ －オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション 遺伝子置換及び抗炎症を介したデュシェンヌ型筋ジストロフィーに対する一段階遺伝子療法
US20220204574A1 (en) * 2019-03-25 2022-06-30 Genethon Production of large-sized quasidystrophins using overlapping aav vectors
CN111088284B (zh) * 2019-05-31 2022-02-01 北京京谱迪基因科技有限公司 携带截短抗肌萎缩蛋白基因表达框的aav载体、制备方法及应用
WO2020261178A1 (en) * 2019-06-27 2020-12-30 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
CN114302968A (zh) * 2019-08-29 2022-04-08 美国西门子医学诊断股份有限公司 用于检测aav脱落的试剂和方法
CN111057157A (zh) * 2020-01-02 2020-04-24 北京辉润合众生物科技有限公司 一种抗肌萎缩融合蛋白mDp116
KR20230003557A (ko) 2020-04-29 2023-01-06 브리스톨-마이어스 스큅 컴퍼니 스펙트린 융합 도메인을 갖는 소형화 디스트로핀 및 그의 용도
AU2021293197A1 (en) * 2020-06-15 2023-02-09 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery for muscular dystrophies
WO2022029543A1 (en) * 2020-08-06 2022-02-10 Intas Pharmaceuticals Ltd. Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
CA3195177A1 (en) * 2020-10-07 2022-04-14 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i)
US20240003898A1 (en) * 2020-10-30 2024-01-04 Pfizer Inc. Methods for measuring dystrophin in tissue samples
MX2023005218A (es) 2020-11-03 2023-05-16 Pfizer Metodos de purificacion de vectores de aav mediante cromatografia de intercambio anionico.
WO2022122733A1 (en) * 2020-12-08 2022-06-16 Genethon New gene therapy for the treatment of duchenne muscular dystrophy
IL303912A (en) 2020-12-21 2023-08-01 Pfizer Methods and preparations for preventing excess sedimentation of nucleic acids
IL303920A (en) 2020-12-21 2023-08-01 Pfizer Methods and systems for improved transfection of cells
AU2021404944A1 (en) 2020-12-23 2023-07-06 Pfizer Inc. Methods for purification of aav vectors by affinity chromatography
CN112860362B (zh) * 2021-02-05 2022-10-04 达而观数据(成都)有限公司 一种机器人自动化流程的可视化调试方法及调试系统
RU2767335C1 (ru) * 2021-03-02 2022-03-17 Общество с ограниченной ответственностью «Марлин Биотех» Химерные белки на основе утрофина и дистрофина человека и их применение для лечения миодистрофии Дюшенна
WO2022229351A1 (en) 2021-04-28 2022-11-03 Graviton Bioscience Bv Selective inhibitors of rock2 for the treatment of muscular dystrophy
EP4108263A3 (en) 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
EP4413018A1 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for targeted delivery
CN114316070B (zh) * 2021-12-29 2022-11-15 上海勉亦生物科技有限公司 用于治疗肌营养不良症的转基因表达盒
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
EP4230196A1 (en) 2022-02-21 2023-08-23 Som Innovation Biotech, S.A. Compounds for use in the treatment of dystrophinopathies
IT202200006119A1 (it) * 2022-03-29 2023-09-29 Univ Degli Studi Di Ferrara Method for implementing the design of synthetic nucleic acid molecules for gene therapies in rare diseases.
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
WO2024138158A2 (en) * 2022-12-23 2024-06-27 Ultragenyx Pharmaceutical Inc. Method of producing soluble microdystrophin protein and uses of the same

Family Cites Families (48)

* 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
RU2219241C2 (ru) * 1993-07-13 2003-12-20 Рон-Пуленк Роре С.А. Дефектный рекомбинантный аденовирусный вектор (варианты)
AU707862B2 (en) 1995-06-07 1999-07-22 University Of North Carolina At Chapel Hill, The 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
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
US5994132A (en) 1996-10-23 1999-11-30 University Of Michigan Adenovirus vectors
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
JPH11318467A (ja) 1998-05-08 1999-11-24 Japan Science & Technology Corp 短縮型ジストロフィン
ATE402254T1 (de) 1998-05-28 2008-08-15 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
PT1127150E (pt) 1998-11-05 2007-08-22 Univ Pennsylvania ''sequências de ácido nucleico do vírus adeno associado do serotipo 1, vectores e células hospedeiras que as contêm''
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
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
EP1287125B1 (en) 2000-04-28 2009-07-29 Asklepios Biopharmaceutical, Inc. Dna sequences encoding dystrophin minigenes and methods of use thereof
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
WO2001092551A2 (en) 2000-06-01 2001-12-06 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
DE60130465T2 (de) 2000-07-14 2008-06-12 The Regents Of The University Of Michigan, Ann Arbor Mutierte muskelspezifische enhancer
US6812339B1 (en) 2000-09-08 2004-11-02 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
WO2002029056A2 (en) 2000-10-06 2002-04-11 Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
WO2002046359A2 (en) 2000-12-07 2002-06-13 Universite De Nantes Inducible highly productive raav packaging cell-lines
WO2002081517A2 (en) 2001-01-19 2002-10-17 Curagen Corporation Novel polypeptides and nucleic acids encoded thereby
US7112668B2 (en) 2001-01-23 2006-09-26 Curagen Corporation Polypeptides and nucleic acids encoded thereby
US20040142325A1 (en) 2001-09-14 2004-07-22 Liat Mintz Methods and systems for annotating biomolecular sequences
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
WO2005049850A2 (en) * 2003-11-14 2005-06-02 University Of Washington Compositions and methods for systemic nucleic acid sequence delivery
US7771993B2 (en) 2004-01-23 2010-08-10 The Trustees Of The University Of Pennsylvania Microutrophin and uses thereof
EP1716227A4 (en) 2004-01-27 2010-01-06 Compugen Ltd METHOD OF IDENTIFYING PUTATIVE GENE PRODUCTS USING THE INTERSPEZIES SEQUENCE COMPARISON AND BIOMOLECULAR SEQUENCES DISCOVERED THEREFOR
ES2659114T3 (es) 2005-08-19 2018-03-13 Wyeth Llc Anticuerpos antagonistas contra GDF-8 y usos en el tratamiento de ALS y otros trastornos asociados con GDF-8
CA2634146A1 (en) 2005-12-19 2007-06-28 Genizon Biosciences Inc. Genemap of the human genes associated with crohn's disease
AU2007284651B2 (en) 2006-08-09 2014-03-20 Institute For Systems Biology Organ-specific proteins and methods of their use
CN101711164B (zh) * 2007-01-18 2014-06-04 密苏里-哥伦比亚大学 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因
WO2008118258A2 (en) 2007-02-06 2008-10-02 Genizon Biosciences Inc. Genemap of the human genes associated with adhd
WO2008112177A2 (en) 2007-03-08 2008-09-18 Genizon Biosciences, Inc. Genemap of the human genes associated with schizophrenia
PE20091163A1 (es) 2007-11-01 2009-08-09 Wyeth Corp Anticuerpos para gdf8
US20140303093A1 (en) 2011-07-12 2014-10-09 Regents Of The University Of Minnesota Micro-utrophin polypeptides and methods
EP2771455B1 (en) 2011-10-28 2016-10-05 The University of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
US20130136729A1 (en) 2011-11-11 2013-05-30 University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors
US9303079B2 (en) 2012-04-02 2016-04-05 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
CA2876397C (en) 2012-06-15 2019-08-06 Pfizer Inc. Improved antagonist antibodies against gdf-8 and uses therefor
US9624282B2 (en) 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
EP3024498B1 (en) 2013-07-22 2019-12-04 The Children's Hospital of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
EP3114232A4 (en) * 2014-03-03 2017-08-09 Acharjee, Sujata Chimeric dystrophin-vsv-g-protein to treat dystrophinopathies
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
KR102684387B1 (ko) 2015-01-16 2024-07-11 유니버시티 오브 워싱톤 신규한 마이크로-디스트로핀 및 관련된 사용 방법
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use

Also Published As

Publication number Publication date
IL305467A (en) 2023-10-01
RU2019101208A3 (pt) 2020-07-21
CA2971303A1 (en) 2017-12-21
JP7239540B2 (ja) 2023-03-14
IL263199A (en) 2018-12-31
US20170368198A1 (en) 2017-12-28
MX2018015921A (es) 2019-08-16
KR20190027358A (ko) 2019-03-14
SG11201811115TA (en) 2019-01-30
US20230398236A1 (en) 2023-12-14
TW201812010A (zh) 2018-04-01
WO2017221145A9 (en) 2019-01-17
EP3472194A1 (en) 2019-04-24
RU2019101208A (ru) 2020-07-21
JP6793758B2 (ja) 2020-12-02
AU2017281983B2 (en) 2022-04-21
AU2017281983A1 (en) 2018-12-13
IL263199B1 (en) 2023-09-01
IL263199B2 (en) 2024-01-01
CN116064555A (zh) 2023-05-05
JP2021045136A (ja) 2021-03-25
PE20190563A1 (es) 2019-04-22
TWI719223B (zh) 2021-02-21
MY197846A (en) 2023-07-20
PH12018502645A1 (en) 2019-10-14
CN109641944A (zh) 2019-04-16
US11547765B2 (en) 2023-01-10
WO2017221145A1 (en) 2017-12-28
RU2753194C2 (ru) 2021-08-12
CO2019000395A2 (es) 2019-04-30
KR102568278B1 (ko) 2023-08-22
CN109641944B (zh) 2022-07-19
JP2019525740A (ja) 2019-09-12
US20200376141A1 (en) 2020-12-03
SA518400623B1 (ar) 2023-12-15

Similar Documents

Publication Publication Date Title
BR112018076394A2 (pt) genes de minidistrofina otimizados e cassetes de expressão e seu uso
BR112018074463A2 (pt) anticorpos anti-tim-3 e métodos de uso dos mesmos.
MX2020004145A (es) Variantes de desoxirribonucleasa (dnasa).
BR112018011881A2 (pt) proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus
CL2018000490A1 (es) Anticuerpos anti-pd1 y sus métodos de uso
AR122050A2 (es) Proteínas de fusión, bacterias recombinantes y métodos de uso de las bacterias recombinantes
BR112019010565A2 (pt) aplicação viral de neoantígenos
BR112017025564A2 (pt) anticorpos anti-ctla-4 e métodos de uso dos mesmos
CO2018002703A2 (es) Anticuerpos anti-promiostatina o miostatina latente y usos de los mismos
BR112019001737A2 (pt) expressão de pten-long com vírus oncolíticos
BR112019000195A2 (pt) composições e métodos relacionados a sistemas celulares terapêuticos que expressam rna exógeno
BR112019005964A2 (pt) proteínas de fusão imunomoduladoras
CL2017002820A1 (es) Variantes de alfa-amilasa y polinucleótidos que codifican las mismas
CL2018001522A1 (es) Focalización de péptidos para dirigir virus adenoasociados (aavs)
UY35964A (es) Anticuerpos humanos para pd?1
BR112018012180A2 (pt) vírus de codificação de um anticorpo ou fragmento de complexo anti-tcr
CO2017006580A2 (es) Anticuerpos humanos para hemaglutinina de influenza
CL2017002731A1 (es) Adenovirus oncolítico que codifica una proteína b7
CO2017005842A2 (es) Anticuerpos heterodiméricos que se unen a cd3 y cd38
BR112016020287A2 (pt) células resistentes a vírus e usos das mesmas
BR112018006237A2 (pt) proteínas de ligação a pd-1 e métodos de uso das mesmas
BR112016026950A2 (pt) composições de rnai para angiotensinogênio (agt) e métodos de uso das mesmas
CR20150437A (es) Construcciones de anticuerpos para cdh19 y cd3
BR112019012342A2 (pt) anticorpos il-11
BR112016016916A8 (pt) uso de um anticorpo que se liga à calicreína plasmática ativa para tratamento de angiodema hereditário (hae)

Legal Events

Date Code Title Description
B350 Update of information on the portal [chapter 15.35 patent gazette]
B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]