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

Info

Publication number
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
Authority
CN
China
Prior art keywords
mir
hsa
mirna
nucleic acid
expression
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
CN201880051148.4A
Other languages
English (en)
Chinese (zh)
Inventor
M·S·埃斯特维斯
高光平
M·R·格林
王丹
T·M·西蒙尼
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 CN111065742A publication Critical patent/CN111065742A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/10—Production naturally occurring
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (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)

Publication Number Publication Date
CN111065742A true CN111065742A (zh) 2020-04-24

Family

ID=64567243

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201880051148.4A Pending CN111065742A (zh) 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

Country Status (15)

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

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 (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
SG10202105090WA (en) * 2016-11-17 2021-06-29 Nationwide Childrens Hospital Inc Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2
US12138298B2 (en) 2017-01-30 2024-11-12 Brainvectis Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
US12091435B2 (en) 2018-04-03 2024-09-17 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
MX2020010464A (es) 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
CN119684417A (zh) 2018-10-02 2025-03-25 沃雅戈治疗公司 重新定向aav衣壳的嗜性
JP7698583B2 (ja) 2019-03-21 2025-06-25 ギンコ バイオワークス インコーポレイテッド 組換えアデノ随伴ウイルスベクター
SG11202111279QA (en) * 2019-04-24 2021-11-29 Univ Pennsylvania Compositions useful in treatment of rett syndrome
CN114555813A (zh) * 2019-05-21 2022-05-27 国家医疗保健研究所 治疗雷特综合征的胆固醇24-水解酶的表达载体
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for treatment of niemann-pick disease type c
IL293431A (en) * 2019-12-05 2022-07-01 Univ Texas Transgene cassettes designed to express human mecp2 gene
US12497612B2 (en) 2020-03-09 2025-12-16 University Of Massachusetts Gene replacement therapy for FOXG1 syndrome
IL298001A (en) * 2020-05-13 2023-01-01 Voyager Therapeutics Inc Redirecting tropism of adeno-associated virus capsids
AU2021300615A1 (en) * 2020-06-30 2023-02-02 The University Court Of The University Of Edinburgh Transgene expression system
CA3189878A1 (en) * 2020-08-19 2022-02-24 Colin O'BANION 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
WO2023081648A1 (en) 2021-11-02 2023-05-11 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
KR20260016905A (ko) * 2023-03-22 2026-02-04 오스페달레 산 라파엘 에스.알.엘. 유전자 치료

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
CN103555677B (zh) 2001-11-13 2018-01-30 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
EA201000603A1 (ru) * 2007-10-23 2010-12-30 Новартис Аг ПРИМЕНЕНИЕ АНТИТЕЛ К TrkB ДЛЯ ЛЕЧЕНИЯ РЕСПИРАТОРНЫХ НАРУШЕНИЙ
CN102015769B (zh) * 2008-01-17 2014-12-10 Irm责任有限公司 改进的抗-trkb抗体
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)
CA2815582A1 (en) 2010-10-25 2012-05-03 Inserm (Institut National De La Sante Et De La Recherche Medicale) 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
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
EP3795580A1 (en) 2014-10-03 2021-03-24 University of Massachusetts High efficiency library-identified aav vectors
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
CA3021949C (en) 2015-04-24 2023-10-17 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

Also Published As

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

Similar Documents

Publication Publication Date Title
CN111065742A (zh) 用于MECP2在Rett综合征中安全表达的自调节AAV载体
AU2023201093B2 (en) Adeno-Associated Virus Vector Delivery Of Micro-Dystrophin To Treat Muscular Dystrophy
KR102135239B1 (ko) 세포 면역요법을 위한 방법 및 조성물
US20190282709A1 (en) Isolation of novel aav's and uses thereof
CN108384784A (zh) 一种利用CRISPR/Cas9技术敲除Endoglin基因的方法
CN109923211A (zh) Pd-1归巢核酸内切酶变体、组合物及使用方法
BR112019019248A2 (pt) entrega de vetor de vírus adeno-associado de microdistrofina específica muscular para tratamento de distrofia muscular
KR20180054886A (ko) 변형된 인간 t 세포 수용체 알파 불변 영역 유전자를 포함하는 유전자 변형된 세포
WO2010138263A2 (en) Novel aav 's and uses thereof
KR20110122672A (ko) 이소프렌 및 공-산물을 제조하는 방법
TW201120213A (en) Polymerization of isoprene from renewable resources
TW202233844A (zh) Aav衣殼及含有其之組成物
AU2023412590A1 (en) Fusion and use thereof
CN114206393A (zh) 用于治疗天使综合征的载体和方法
US7090836B2 (en) Vector for expressing α-L-iduronidase and method of treating MPS I by stereotactic injection into the brain of a mammal
CN115698332A (zh) 在单细胞水平上评估载体转导效率和/或特异性的方法
AU2018326582B2 (en) Retinal promoter and uses thereof
ES2791877T3 (es) Vectores AAV para terapia génica vascular en cardiopatía coronaria e isquemia periférica
CN111298129A (zh) 二甲双胍介导核酸纳米材料自组装方法及采用该方法制备的纳米制剂和应用
EA047963B1 (ru) Вектор и способ для лечения синдрома ангельмана
CN111388658A (zh) 基于重组减毒李斯特菌的kras高表达癌症疫苗及其制备方法及其应用方法

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

Application publication date: 20200424

WD01 Invention patent application deemed withdrawn after publication