CN111065742A - 用于MECP2在Rett综合征中安全表达的自调节AAV载体 - Google Patents

用于MECP2在Rett综合征中安全表达的自调节AAV载体 Download PDF

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CN111065742A
CN111065742A CN201880051148.4A CN201880051148A CN111065742A CN 111065742 A CN111065742 A CN 111065742A CN 201880051148 A CN201880051148 A CN 201880051148A CN 111065742 A CN111065742 A CN 111065742A
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mir
hsa
mirna
nucleic acid
expression
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M·S·埃斯特维斯
高光平
M·R·格林
王丹
T·M·西蒙尼
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University of Massachusetts Amherst
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    • 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
    • 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
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/41—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2330/00—Production
    • C12N2330/10—Production naturally occurring
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN201880051148.4A 2017-06-06 2018-06-06 用于MECP2在Rett综合征中安全表达的自调节AAV载体 Pending CN111065742A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762516060P 2017-06-06 2017-06-06
US62/516,060 2017-06-06
PCT/US2018/036200 WO2018226785A1 (en) 2017-06-06 2018-06-06 Self-regulating aav vectors for safe expression of mecp2 in rett syndrome

Publications (1)

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CN111065742A true CN111065742A (zh) 2020-04-24

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US (2) US11680275B2 (https=)
EP (1) EP3635122A4 (https=)
JP (2) JP2020522269A (https=)
KR (1) KR20200015701A (https=)
CN (1) CN111065742A (https=)
AU (1) AU2018281145A1 (https=)
BR (1) BR112019025732A2 (https=)
CA (1) CA3066623A1 (https=)
CL (1) CL2019003582A1 (https=)
CO (1) CO2019014840A2 (https=)
EA (1) EA201992882A1 (https=)
IL (1) IL271170A (https=)
MX (1) MX2019014760A (https=)
SG (1) SG11201911737PA (https=)
WO (1) WO2018226785A1 (https=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113754727A (zh) * 2020-09-30 2021-12-07 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024235164A1 (zh) * 2023-05-15 2024-11-21 上海金珂博生物技术有限公司 一种Rett综合症的基因治疗
WO2024251232A1 (zh) * 2023-06-09 2024-12-12 仁景(苏州)生物科技有限公司 表达可调控的工程化rna分子

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
JP7106534B2 (ja) * 2016-11-17 2022-07-26 ネイションワイド チルドレンズ ホスピタル， インコーポレイテッド メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達
JP7618386B2 (ja) 2017-01-30 2025-01-21 ブレインヴェクティス ポリグルタミン反復脊髄小脳失調症の処置におけるコレステロール２４－ヒドロキシラーゼ（ｈｙｄｒｏｌａｓｅ）のための発現ベクター
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
CA3133889A1 (en) * 2019-04-24 2020-10-29 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
JP7624410B2 (ja) * 2019-05-21 2025-01-30 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル レット症候群の治療におけるコレステロール ２４－ヒドロラーゼのための発現ベクター
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
EP4065600A1 (en) 2019-11-25 2022-10-05 Cornell University Apoe gene therapy
JP7702949B2 (ja) * 2019-12-05 2025-07-04 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム ヒトｍｅｃｐ２遺伝子を発現するように設計されたトランスジーンカセット
US12497612B2 (en) 2020-03-09 2025-12-16 University Of Massachusetts Gene replacement therapy for FOXG1 syndrome
MX2022014258A (es) * 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones para la reducción específica de un fármaco de la expresión de transgén.
TWI900553B (zh) 2020-05-13 2025-10-11 美商航海家醫療公司 Aav蛋白殼之趨性重定向
MX2023000124A (es) * 2020-06-30 2023-03-08 Univ Court Univ Of Edinburgh Sistema de expresion de transgenes.
AU2021328475A1 (en) * 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
WO2022094078A1 (en) * 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
GB202201242D0 (en) * 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
CN121219020A (zh) * 2023-03-22 2025-12-26 圣拉斐尔医院有限责任公司 基因疗法

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040106566A1 (en) * 2002-05-17 2004-06-03 Shi-Lung Lin RNA-splicing and processing-directed gene silencing and the relative applications thereof
WO2015164786A1 (en) * 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
EP3121284A1 (en) * 2015-07-22 2017-01-25 Ecole Polytechnique Fédérale de Lausanne (EPFL) Bicistronic aav vector for rna interference in als

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
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.
EP2214706A1 (en) 2007-10-23 2010-08-11 Novartis AG Use of trkb antibodies for the treatment of respiratory disorders
PE20091326A1 (es) 2008-01-17 2009-09-18 Irm Llc Anticuerpos anti-trkb mejorados
WO2010105096A2 (en) 2009-03-11 2010-09-16 University Of Massachusetts Modulation of human cytomegalovirus replication by micro-rna 132 (mir132), micro-rna 145 (mir145) and micro-rna 212 (mir212)
WO2012055826A1 (en) 2010-10-25 2012-05-03 Universite De La Mediterranee (Aix-Marseille Ii) TREATMENT OF MeCP2-ASSOCIATED DISORDERS
US20160000794A1 (en) 2012-09-25 2016-01-07 The United States Of America, As Represented By The Secretary, Department Of Health And Human Serv Methods for the diagnosis and treatment of sjogren's syndrome
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3200830B1 (en) 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040106566A1 (en) * 2002-05-17 2004-06-03 Shi-Lung Lin RNA-splicing and processing-directed gene silencing and the relative applications thereof
WO2015164786A1 (en) * 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
EP3121284A1 (en) * 2015-07-22 2017-01-25 Ecole Polytechnique Fédérale de Lausanne (EPFL) Bicistronic aav vector for rna interference in als

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
GADALLA ET AL.: "Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome", 《MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT》 *
GADALLA ET AL.: "Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome", 《MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT》, 22 April 2017 (2017-04-22) *
MATTHEW E KLEIN ET AL: "Homeostatic regulation of MeCP2 expression by a CREB-induced microRNA", 《NATURE NEUROSCIENCE》, pages 2 - 3 *
SINNET ET AL.: "Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery", 《MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT》, 19 April 2017 (2017-04-19), pages 112 *
SINNET ET AL: "Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery", 《MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT》, pages 112 *
XIE ET AL.: "MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression", 《MOLECULAR THERAPY》, 19 November 2010 (2010-11-19) *
XIE ET AL: "MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression", 《MOLECULAR THERAPY》, pages 527 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113754727A (zh) * 2020-09-30 2021-12-07 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024235164A1 (zh) * 2023-05-15 2024-11-21 上海金珂博生物技术有限公司 一种Rett综合症的基因治疗
WO2024251232A1 (zh) * 2023-06-09 2024-12-12 仁景(苏州)生物科技有限公司 表达可调控的工程化rna分子

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JP2023164447A (ja) 2023-11-10
CL2019003582A1 (es) 2020-09-25
EP3635122A4 (en) 2021-03-31
CO2019014840A2 (es) 2020-01-17
MX2019014760A (es) 2020-08-03
KR20200015701A (ko) 2020-02-12
EA201992882A1 (ru) 2020-05-25
US20200181646A1 (en) 2020-06-11
JP2020522269A (ja) 2020-07-30
BR112019025732A2 (pt) 2020-06-30
US11680275B2 (en) 2023-06-20
EP3635122A1 (en) 2020-04-15
AU2018281145A1 (en) 2020-01-02
SG11201911737PA (en) 2020-01-30
WO2018226785A1 (en) 2018-12-13
IL271170A (en) 2020-01-30
CA3066623A1 (en) 2019-12-13
US20230416779A1 (en) 2023-12-28

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