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

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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
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bone
gly
nucleic acid
pro
raav
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沈载赫
高光平
谢军
金瀞慜
王丹
梁软淑
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University of Massachusetts Amherst
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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

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CN112368051A true CN112368051A (zh) 2021-02-12

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

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US (2) US12194108B2 (https=)
EP (2) EP4169576A1 (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
EP4169576A1 (en) 2018-03-23 2023-04-26 University of Massachusetts Gene therapeutics for treating bone disorders
US20240002844A1 (en) * 2020-08-19 2024-01-04 University Of Massachusetts Development of novel gene therapeutics for 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
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania 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
ATE481487T1 (de) 1998-11-27 2010-10-15 Ucb Sa Zusammensetzungen und verfahren zur erhöhung der knochenmineralisierung
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP1874811B1 (en) 2005-04-15 2012-06-13 President and Fellows of Harvard College Methods for modulating bone formation and mineralization by modulating KRC activity
US8859752B2 (en) 2006-04-18 2014-10-14 The Trustees Of The University Of Pennsylvania SIRNA-based therapy of Fibrodyplasia Ossificans Progressiva (FOP)
WO2007127428A2 (en) 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
WO2008063839A2 (en) 2006-10-31 2008-05-29 University Of Rochester Targeted delivery of therapeutic agents with lyophilized matrices
CA2683314A1 (en) 2007-04-11 2008-10-23 Merck & Co., Inc. Isolated nucleic acid molecules corresponding to micro rna 145 (mirna-145) and their use in treating colon cancer
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
KR20140013887A (ko) 2010-05-03 2014-02-05 더 텍사스 에이 & 엠 유니버시티 시스템 눈 손상 및 질환 치료용 성체 줄기 세포/전구 세포 및 줄기 세포 단백질
CA2815181C (en) 2010-10-27 2020-09-15 William Gleason Richards Dkk1 antibodies and methods of use
CN107115352B (zh) 2011-08-04 2020-10-30 耶达研究及发展有限公司 微rna和包含微rna的组合物
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics 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
JP6601687B2 (ja) 2014-03-31 2019-11-06 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
WO2015183667A1 (en) 2014-05-28 2015-12-03 The Regents Of The University Of California HYBRID tRNA/pre-miRNA MOLECULES AND METHODS OF USE
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
TWI632160B (zh) * 2015-01-16 2018-08-11 中央研究院 具有標的部分及效應部分的分子構建體
EA201791630A1 (ru) 2015-01-20 2018-04-30 Джензим Корпорейшн Аналитическое ультрацентрифугирование для определения характеристик рекомбинантных вирусных частиц
CR20170406A (es) 2015-02-10 2017-11-14 Genzyme Corp Arni variante
EP3256170B1 (en) 2015-02-13 2020-09-23 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
US10526657B2 (en) 2015-12-10 2020-01-07 Universitat Fur Bodenkultur Wien Compositions and methods for the diagnosis and treatment of bone fractures and disorders
WO2017191274A2 (en) 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
WO2018078303A1 (fr) 2016-10-27 2018-05-03 Selexel Nouvel oligonucleotide double brin bisquecifiques pour le traitement des cancers
KR102719587B1 (ko) 2017-02-17 2024-10-17 론자 리미티드 아데노-관련 바이러스 생산을 위한 포유동물 세포
EP4169576A1 (en) 2018-03-23 2023-04-26 University of Massachusetts Gene therapeutics for treating bone disorders
WO2020118011A1 (en) 2018-12-06 2020-06-11 Alexion Pharmaceuticals, Inc. Anti-alk2 antibodies and uses thereof
EP3891175A4 (en) 2018-12-06 2022-11-09 Arcturus Therapeutics, Inc. MODIFIED PROTEINS AND RELATED TREATMENT METHODS
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
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
US20240002844A1 (en) 2020-08-19 2024-01-04 University Of Massachusetts Development of novel gene therapeutics for inflammation-induced bone loss
KR20230117177A (ko) 2020-12-03 2023-08-07 유니버시티 오브 매사추세츠 진행성 골화성 섬유이형성증을 위한 신규 유전자 치료제의개발
WO2022221581A1 (en) 2021-04-15 2022-10-20 Mammoth Biosciences, Inc. Programmable nucleases and methods of use
US20240382513A1 (en) 2021-07-09 2024-11-21 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health

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

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