WO2016106458A8 - Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimización y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje - Google Patents

Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimización y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje Download PDF

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Publication number
WO2016106458A8
WO2016106458A8 PCT/CL2015/000069 CL2015000069W WO2016106458A8 WO 2016106458 A8 WO2016106458 A8 WO 2016106458A8 CL 2015000069 W CL2015000069 W CL 2015000069W WO 2016106458 A8 WO2016106458 A8 WO 2016106458A8
Authority
WO
WIPO (PCT)
Prior art keywords
xbp1s
aav
virus
learning
improvement
Prior art date
Application number
PCT/CL2015/000069
Other languages
English (en)
French (fr)
Other versions
WO2016106458A1 (es
Inventor
Claudio Andrés HETZ FLORES
Gabriela Raquel Elena MARTINEZ BRAVO
Original Assignee
Universidad De Chile
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 Universidad De Chile filed Critical Universidad De Chile
Priority to US15/539,826 priority Critical patent/US11400166B2/en
Priority to EP15874441.7A priority patent/EP3254702B1/en
Priority to PL15874441.7T priority patent/PL3254702T3/pl
Priority to ES15874441T priority patent/ES2949828T3/es
Publication of WO2016106458A1 publication Critical patent/WO2016106458A1/es
Priority to US16/858,435 priority patent/US20210060178A1/en
Publication of WO2016106458A8 publication Critical patent/WO2016106458A8/es

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    • 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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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

Abstract

La presente invención presenta una secuencia del virus AAV/XBPls-HA, método y su uso en el mejoramiento de funciones cognitivas, de memoria y del aprendizaje, tal como se presenta en los estudios in vivo en la figura 12/17 panel derecho.
PCT/CL2015/000069 2014-12-30 2015-12-24 Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimización y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje WO2016106458A1 (es)

Priority Applications (5)

Application Number Priority Date Filing Date Title
US15/539,826 US11400166B2 (en) 2014-12-30 2015-12-24 AAV/XBP1s-HA virus, gene therapy method and use thereof in the optimisation and improvement of learning, memory and cognitive capacities
EP15874441.7A EP3254702B1 (en) 2014-12-30 2015-12-24 Aav/xbp1s-ha virus, gene therapy method and use thereof in the optimisation and improvement of learning, memory and cognitive capacities
PL15874441.7T PL3254702T3 (pl) 2014-12-30 2015-12-24 Wirus aav/xbp1s-ha, sposób terapii genowej i jego stosowanie w optymalizacji i poprawie zdolności uczenia się, pamięci i zdolności poznawczych
ES15874441T ES2949828T3 (es) 2014-12-30 2015-12-24 Virus AAV/XBP1S-HA, método de terapia génica y uso de este en la optimización y mejora de las capacidades de aprendizaje, memoria y cognitivas
US16/858,435 US20210060178A1 (en) 2014-12-30 2020-04-24 Aav/xbp1s-ha virus, gene therapy method and use thereof in the optimisation and improvement of learning, memory and cognitive capacities

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CL2014003590A CL2014003590A1 (es) 2014-12-30 2014-12-30 Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimizacion y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje.
CL3590-2014 2014-12-30

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US15/539,826 A-371-Of-International US11400166B2 (en) 2014-12-30 2015-12-24 AAV/XBP1s-HA virus, gene therapy method and use thereof in the optimisation and improvement of learning, memory and cognitive capacities
US16/858,435 Division US20210060178A1 (en) 2014-12-30 2020-04-24 Aav/xbp1s-ha virus, gene therapy method and use thereof in the optimisation and improvement of learning, memory and cognitive capacities

Publications (2)

Publication Number Publication Date
WO2016106458A1 WO2016106458A1 (es) 2016-07-07
WO2016106458A8 true WO2016106458A8 (es) 2021-12-23

Family

ID=56283749

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/CL2015/000069 WO2016106458A1 (es) 2014-12-30 2015-12-24 Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimización y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje

Country Status (6)

Country Link
US (2) US11400166B2 (es)
EP (1) EP3254702B1 (es)
CL (1) CL2014003590A1 (es)
ES (1) ES2949828T3 (es)
PL (1) PL3254702T3 (es)
WO (1) WO2016106458A1 (es)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CL2015003024A1 (es) * 2015-10-09 2016-05-13 Univ Chile Método de tratamiento genético utilizando el virus aav-xbp1s/gfp, y su uso en la prevención y tratamiento de la esclerosis lateral amiotrofica.
CL2015003242A1 (es) * 2015-11-04 2016-10-14 Univ Chile Virus aav/upr-plus, proteína de fusión upr-plus, método de tratamiento genético y su uso en el tratamiento enfermedades neurodegenerativas, tales como las enfermedades de parkinson y huntington, entre otras.
CL2016003282A1 (es) * 2016-12-21 2017-08-18 Univ Chile Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington
WO2020157700A1 (en) * 2019-02-01 2020-08-06 Universidad De Chile Treatment of aging or age-related disorders using xbp1
TW202115126A (zh) 2019-06-11 2021-04-16 美商舒爾人類基因療法公司 用於治療血漿激肽釋放酶失調所介導之疾病的抗體腺相關病毒載體遞送
US20230124994A1 (en) * 2019-07-18 2023-04-20 Lysogene Compositions and methods for the treatment of sanfilippo disease and other disorders
WO2021081280A1 (en) 2019-10-23 2021-04-29 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema
CN116829595A (zh) 2020-12-16 2023-09-29 武田药品工业株式会社 用于治疗由失调的血浆激肽释放酶介导的疾病的抗体的腺相关病毒载体递送
WO2023143435A1 (zh) * 2022-01-29 2023-08-03 上海日馨医药科技股份有限公司 表达tpk的重组病毒及其治疗阿尔茨海默病的用途
CN115779083B (zh) * 2022-08-05 2024-04-02 河南省精神病医院(新乡医学院第二附属医院) 下调Tp73蛋白表达的物质在制备治疗酒依赖的药物中的应用

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7090836B2 (en) 2002-06-21 2006-08-15 Institut Pasteur Vector for expressing α-L-iduronidase and method of treating MPS I by stereotactic injection into the brain of a mammal
US8198257B2 (en) * 2007-09-12 2012-06-12 Institut National De La Sante Et De La Recherche Medicale (Inserm) CYP46A1 gene for the treatment of alzheimer's disease

Also Published As

Publication number Publication date
EP3254702A1 (en) 2017-12-13
PL3254702T3 (pl) 2023-09-11
US20170360961A1 (en) 2017-12-21
CL2014003590A1 (es) 2015-07-10
US20210060178A1 (en) 2021-03-04
ES2949828T3 (es) 2023-10-03
EP3254702B1 (en) 2023-05-10
WO2016106458A1 (es) 2016-07-07
US11400166B2 (en) 2022-08-02
EP3254702A4 (en) 2019-02-20

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