CN112368051A - 用于治疗骨病的基因治疗剂 - Google Patents

用于治疗骨病的基因治疗剂 Download PDF

Info

Publication number
CN112368051A
CN112368051A CN201980031173.0A CN201980031173A CN112368051A CN 112368051 A CN112368051 A CN 112368051A CN 201980031173 A CN201980031173 A CN 201980031173A CN 112368051 A CN112368051 A CN 112368051A
Authority
CN
China
Prior art keywords
bone
gly
nucleic acid
pro
raav
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN201980031173.0A
Other languages
English (en)
Chinese (zh)
Inventor
沈载赫
高光平
谢军
金瀞慜
王丹
梁软淑
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Massachusetts Amherst
Original Assignee
University of Massachusetts Amherst
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 University of Massachusetts Amherst filed Critical University of Massachusetts Amherst
Publication of CN112368051A publication Critical patent/CN112368051A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575Hormones
    • C07K14/635Parathyroid hormone, i.e. parathormone; Parathyroid hormone-related peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/035Animal model for multifactorial diseases
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Endocrinology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (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)
  • Materials For Medical Uses (AREA)
CN201980031173.0A 2018-03-23 2019-03-22 用于治疗骨病的基因治疗剂 Pending CN112368051A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862647595P 2018-03-23 2018-03-23
US62/647,595 2018-03-23
US201962799843P 2019-02-01 2019-02-01
US62/799,843 2019-02-01
PCT/US2019/023759 WO2019183605A1 (en) 2018-03-23 2019-03-22 Gene therapeutics for treating bone disorders

Publications (1)

Publication Number Publication Date
CN112368051A true CN112368051A (zh) 2021-02-12

Family

ID=67986628

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980031173.0A Pending CN112368051A (zh) 2018-03-23 2019-03-22 用于治疗骨病的基因治疗剂

Country Status (8)

Country Link
US (2) US12194108B2 (https=)
EP (2) EP3768386A4 (https=)
JP (1) JP2021519065A (https=)
KR (1) KR20200135433A (https=)
CN (1) CN112368051A (https=)
AU (1) AU2019237514A1 (https=)
CA (1) CA3094217A1 (https=)
WO (1) WO2019183605A1 (https=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113913463A (zh) * 2021-09-19 2022-01-11 郭保生 抑制sost基因表达的重组质粒及其骨靶向重组腺相关病毒与应用
CN118453845A (zh) * 2024-05-30 2024-08-09 山东第一医科大学附属省立医院(山东省立医院) 提高dj-1基因表达量的试剂在制备抗血管钙化药物中的应用
CN121249673A (zh) * 2025-12-05 2026-01-02 北京大学第三医院(北京大学第三临床医学院) 一种靶向ybx1的骨关节炎治疗与诊断产品及应用

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019237514A1 (en) 2018-03-23 2020-10-08 University Of Massachusetts Gene therapeutics for treating bone disorders
WO2022040275A1 (en) * 2020-08-19 2022-02-24 University Of Massachusetts Development of novel gene therapeutics inflammation-induced bone loss
US20250388925A1 (en) * 2021-11-04 2025-12-25 University Of Massachusetts Wnt-modulating gene silencers as bone anabolic therapy for osteoporosis and critical-sized bone defect
WO2024220463A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav5 capsid with non-canonical amino acid incorporation and uses thereof
CN120535659B (zh) * 2025-07-29 2025-10-17 苏州大学 一种骨靶向的三聚体蛋白及其制备方法与应用

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100008979A1 (en) * 2004-06-10 2010-01-14 Saint Louis University Delivery of therapeutic agents to the bone
US20100239503A1 (en) * 2009-02-06 2010-09-23 The Regents Of The University Of California Calcium-binding agents induce hair growth and/or nail growth
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
US20130202533A1 (en) * 2010-01-14 2013-08-08 President And Fellows Of Harvard College Methods for modulating skeletal remodeling and patterning by modulating shn2 activity, shn3 activity, or shn2 and shn3 activity in combination
US20140256917A1 (en) * 2005-06-14 2014-09-11 The United States Government As Represented By The Department Of Veterans Affairs Compositions and methods for osteogenic gene therapy
CN107073051A (zh) * 2014-10-21 2017-08-18 马萨诸塞大学 重组aav变体及其用途
WO2018027329A1 (en) * 2016-08-11 2018-02-15 Precithera, Inc. TGF-β ANTAGONIST CONJUGATES
WO2018035503A1 (en) * 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system

Family Cites Families (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5942496A (en) 1994-02-18 1999-08-24 The Regent Of The University Of Michigan Methods and compositions for multiple gene transfer into bone cells
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
DE69933044T3 (de) 1998-11-27 2016-11-24 Ucb Pharma, S.A. Zusammensetzungen und verfahren zur erhöhung der knochenmineralisierung
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
JP2008536869A (ja) 2005-04-15 2008-09-11 プレジデント・アンド・フエローズ・オブ・ハーバード・カレツジ Krc活性を調節することによる骨形成および石灰化の調節方法
US8859752B2 (en) 2006-04-18 2014-10-14 The Trustees Of The University Of Pennsylvania SIRNA-based therapy of Fibrodyplasia Ossificans Progressiva (FOP)
US8298818B2 (en) 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
US8632797B2 (en) 2006-10-31 2014-01-21 University Of Rochester Targeted delivery of therapeutic agents with lyophilized matrices
US20100113577A1 (en) 2007-04-11 2010-05-06 Bin Shi Isolated nucleic acid molecules corresponding to micro rna 145 (mirna-145) and their use in treating colon cancer
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
EP2566566B1 (en) 2010-05-03 2017-09-06 The Texas A&M University System Adult stem cells/progenitor cells and stem cell proteins for treatment of eye injuries and diseases
JP6066912B2 (ja) 2010-10-27 2017-01-25 アムジエン・インコーポレーテツド Dkk1抗体およびその使用方法
US9150858B2 (en) 2011-08-04 2015-10-06 Yeda Research And Development Co. Ltd. Micro-RNAs and compositions comprising same for the treatment and diagnosis of serotonin-, adrenalin-, noradrenalin-, glutamate-, and corticotropin-releasing hormone- associated medical conditions
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
WO2014005314A1 (zh) 2012-07-05 2014-01-09 香港中文大学 基于小核酸药物成骨治疗的骨靶向递送系统及其制备方法
US10143725B2 (en) 2013-03-15 2018-12-04 Biomet Biologics, Llc Treatment of pain using protein solutions
DE102013111099B4 (de) 2013-10-08 2023-11-30 Eberhard Karls Universität Tübingen Medizinische Fakultät Permanente Genkorrektur mittels nukleotidmodifizierter messenger RNA
WO2015152183A1 (ja) 2014-03-31 2015-10-08 大日本住友製薬株式会社 進行性骨化性線維異形成症の予防剤及び治療剤
EP3160980B1 (en) 2014-05-28 2020-05-20 The Regents of the University of California HYBRID tRNA/pre-miRNA MOLECULES AND METHODS OF USE
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
CA2968141C (en) * 2015-01-16 2020-08-04 Hsing-Mao CHU Molecular constructs with targeting and effector elements
CA3301261A1 (en) 2015-01-20 2026-03-02 Genzyme Corporation Analytical ultracentrifugation for characterization of recombinant viral particles
TN2017000354A1 (en) 2015-02-10 2019-01-16 Genzyme Corp VARIANT RNAi
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
JP6805174B2 (ja) * 2015-05-12 2020-12-23 アメリカ合衆国 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むaav分離株及び融合タンパク質
EP3387151A1 (en) 2015-12-10 2018-10-17 Universität für Bodenkultur Wien Compositions and methods for the diagnosis and treatment of bone fractures and disorders
EP3452101A2 (en) 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
JP2020500217A (ja) 2016-10-27 2020-01-09 セレクセルSelexel 癌治療用の新しい二本鎖オリゴヌクレオチド
WO2018150271A1 (en) 2017-02-17 2018-08-23 Lonza Ltd. Mammalian cells for producing adeno-associated viruses
AU2019237514A1 (en) 2018-03-23 2020-10-08 University Of Massachusetts Gene therapeutics for treating bone disorders
JP2022520148A (ja) 2018-12-06 2022-03-29 アークトゥラス・セラピューティクス・インコーポレイテッド 修飾タンパク質および関連の処置方法
US12428484B2 (en) 2018-12-06 2025-09-30 Alexion Pharmaceuticals, Inc. Anti-ALK2 antibodies and uses thereof
CN111407758A (zh) 2019-01-08 2020-07-14 上海中医药大学附属龙华医院 斑蝥素作为miR-214-3p/Wnt/β-Catenin信号通路抑制剂防治骨肉瘤的用途
WO2023283531A2 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health
CA3163283A1 (en) 2020-01-10 2021-07-15 Romesh R. Subramanian Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health
WO2021202494A1 (en) 2020-03-31 2021-10-07 University Of Massachusetts Aav capsids variants and uses thereof
WO2022040275A1 (en) 2020-08-19 2022-02-24 University Of Massachusetts Development of novel gene therapeutics inflammation-induced bone loss
EP4255457A4 (en) 2020-12-03 2025-05-21 University of Massachusetts DEVELOPMENT OF NEW GENE THERAPEUTIC AGENTS FOR FIBRODYSPLASIA OSSIFICANS PROGRESSIVA
WO2022221581A1 (en) 2021-04-15 2022-10-20 Mammoth Biosciences, Inc. Programmable nucleases and methods of use

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100008979A1 (en) * 2004-06-10 2010-01-14 Saint Louis University Delivery of therapeutic agents to the bone
US20140256917A1 (en) * 2005-06-14 2014-09-11 The United States Government As Represented By The Department Of Veterans Affairs Compositions and methods for osteogenic gene therapy
US20100239503A1 (en) * 2009-02-06 2010-09-23 The Regents Of The University Of California Calcium-binding agents induce hair growth and/or nail growth
US20130202533A1 (en) * 2010-01-14 2013-08-08 President And Fellows Of Harvard College Methods for modulating skeletal remodeling and patterning by modulating shn2 activity, shn3 activity, or shn2 and shn3 activity in combination
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
CN107073051A (zh) * 2014-10-21 2017-08-18 马萨诸塞大学 重组aav变体及其用途
WO2018027329A1 (en) * 2016-08-11 2018-02-15 Precithera, Inc. TGF-β ANTAGONIST CONJUGATES
WO2018035503A1 (en) * 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
TAE-KYUNG RYU 等: "Bone-targeted delivery of nanodiamond-based drug carriers conjugated with alendronate for potential osteoporosis treatment", JOURNAL OF CONTROLLED RELEASE, vol. 232, pages 152 - 160, XP029565150, DOI: 10.1016/j.jconrel.2016.04.025 *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113913463A (zh) * 2021-09-19 2022-01-11 郭保生 抑制sost基因表达的重组质粒及其骨靶向重组腺相关病毒与应用
CN113913463B (zh) * 2021-09-19 2023-08-18 郭保生 抑制sost基因表达的重组质粒及其骨靶向重组腺相关病毒与应用
CN118453845A (zh) * 2024-05-30 2024-08-09 山东第一医科大学附属省立医院(山东省立医院) 提高dj-1基因表达量的试剂在制备抗血管钙化药物中的应用
CN121249673A (zh) * 2025-12-05 2026-01-02 北京大学第三医院(北京大学第三临床医学院) 一种靶向ybx1的骨关节炎治疗与诊断产品及应用

Also Published As

Publication number Publication date
AU2019237514A1 (en) 2020-10-08
KR20200135433A (ko) 2020-12-02
US20250170274A1 (en) 2025-05-29
US20210023241A1 (en) 2021-01-28
US12194108B2 (en) 2025-01-14
EP3768386A4 (en) 2022-04-13
CA3094217A1 (en) 2019-09-26
WO2019183605A1 (en) 2019-09-26
EP4169576A1 (en) 2023-04-26
JP2021519065A (ja) 2021-08-10
EP3768386A1 (en) 2021-01-27

Similar Documents

Publication Publication Date Title
US20250170274A1 (en) Gene therapeutics for treating bone disorders
Yang et al. Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis
CN116194154A (zh) 使用aav载体的plakophilin-2(pkp2)基因疗法
JP2024045321A (ja) 合成肝臓指向性アデノ随伴ウイルスカプシドおよびその使用
KR20170086537A (ko) 중추 신경계에 표적화된 aav 벡터
KR20200098481A (ko) Aav 벡터
CN113710693B (zh) Dna结合结构域反式激活因子及其用途
WO2022040275A1 (en) Development of novel gene therapeutics inflammation-induced bone loss
WO2022061378A2 (en) Methods for treating neurological disease
KR20170098931A (ko) 뇌 질환을 치료하기 위한 방법 및 조성물
JP2023510784A (ja) 脳腫瘍を処置するための血液脳関門を横断する免疫療法剤を送達するための方法および組成物
KR20230127269A (ko) 센스, 억제자 트랜스퍼 rna 조성물 및 관련된 용도와기능
WO2020176732A1 (en) TREATMENT OF PULMONARY FIBROSIS WITH SERCA2a GENE THERAPY
US20250388925A1 (en) Wnt-modulating gene silencers as bone anabolic therapy for osteoporosis and critical-sized bone defect
US20260028624A1 (en) Aav-mediated delivery of osteoblast/osteoclast-regulating mirnas for osteoporosis therapy
US20250327080A1 (en) Rnas targeting activin a subunits
JP2022022698A (ja) Aav9を含む骨疾患の予防または治療用の薬学的組成物およびその利用
WO2025174823A1 (en) Engineering of a safe bone anabolic gene therapy to treat alveolar bone loss in osteoporosis
WO2024097521A2 (en) Compositions for treatment of osteogenesis imperfecta
KR20210131236A (ko) Aav9을 포함하는 골 질환 예방 또는 치료용 약학적 조성물 및 그의 이용
HK40100119A (zh) 治疗神经系统疾病的方法
JP2023513211A (ja) タンパク質合成を増強するためのCRISPR-Cas13による標的RNA翻訳
HK40048077A (en) Synthetic liver-tropic adeno-associated virus capsids and uses thereof
Ke Transgenic stem cells for craniofacial bone reconstruction
Morine et al. Systemic Myostatin Inhibition via Liver-Targeted Gene Transfer in Normal and

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
WD01 Invention patent application deemed withdrawn after publication
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20210212