MX2023002978A - Virus adenoasociado recombinante (raav) que codifica la union gap beta 2 (gjb2) y sus usos. - Google Patents

Virus adenoasociado recombinante (raav) que codifica la union gap beta 2 (gjb2) y sus usos.

Info

Publication number
MX2023002978A
MX2023002978A MX2023002978A MX2023002978A MX2023002978A MX 2023002978 A MX2023002978 A MX 2023002978A MX 2023002978 A MX2023002978 A MX 2023002978A MX 2023002978 A MX2023002978 A MX 2023002978A MX 2023002978 A MX2023002978 A MX 2023002978A
Authority
MX
Mexico
Prior art keywords
gjb2
encoding
raav
associated virus
utr
Prior art date
Application number
MX2023002978A
Other languages
English (en)
Inventor
David P Corey
Kevin T Booth
Cole W D Peters
Maryna V Ivanchenko
Michael E Greenberg
Sinisa Hrvatin
Mark Aurel Nagy
Eric C Griffith
Original Assignee
Harvard College
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 Harvard College filed Critical Harvard College
Publication of MX2023002978A publication Critical patent/MX2023002978A/es

Links

Classifications

    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • 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
    • 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
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La presente descripción, al menos en parte, se refiere a composiciones (por ejemplo, ácido nucleico aislado y rAAV) y métodos para tratar la sordera y la pérdida auditiva no sindrómica (DFNB1) mediante la administración de la proteína de la unión gap beta 2 (GJB2) a las células del oído interno que normalmente expresan GJB2 (por ejemplo, fibrocitos y células de soporte del órgano de Corti y regiones cercanas); el ácido nucleico aislado de la presente descripción comprende un casete de expresión, en el que el casete de expresión comprende un elemento regulador del gen (GRE) unión gap beta 2 (GJB2) (por ejemplo, potenciadores de GJB2, promotores de GJB2, GJB2 5' UTR y/o GJB2 3' UTR), y una secuencia de nucleótidos que codifica una proteína GJB2.
MX2023002978A 2020-09-14 2021-09-14 Virus adenoasociado recombinante (raav) que codifica la union gap beta 2 (gjb2) y sus usos. MX2023002978A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063078233P 2020-09-14 2020-09-14
US202163161619P 2021-03-16 2021-03-16
PCT/US2021/050205 WO2022056444A1 (en) 2020-09-14 2021-09-14 Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof

Publications (1)

Publication Number Publication Date
MX2023002978A true MX2023002978A (es) 2023-06-01

Family

ID=80631939

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023002978A MX2023002978A (es) 2020-09-14 2021-09-14 Virus adenoasociado recombinante (raav) que codifica la union gap beta 2 (gjb2) y sus usos.

Country Status (10)

Country Link
EP (1) EP4211151A1 (es)
JP (1) JP2023541443A (es)
KR (1) KR20230069157A (es)
AU (1) AU2021339843A1 (es)
BR (1) BR112023004605A2 (es)
CA (1) CA3191533A1 (es)
IL (1) IL301057A (es)
MX (1) MX2023002978A (es)
TW (1) TW202227476A (es)
WO (1) WO2022056444A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20220133854A (ko) * 2019-09-30 2022-10-05 어플라이드 제네틱스 테크놀로지스 코퍼레이션 유전적 청력 상실 치료를 위한 아데노 관련 바이러스(aav) 시스템
WO2024011224A2 (en) * 2022-07-08 2024-01-11 The Trustees Of Columbia University In The City Of New York Regulatory element for cell type specific expression of genes in spinal motor neurons

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE602005019174D1 (de) * 2004-06-17 2010-03-18 Epigenomics Ag Zusammensetzungen und verfahren zur verhinderung des mitschleppens einer kontamination in nukleinsäure-amplifikationsreaktionen
US20090270480A1 (en) * 2005-12-28 2009-10-29 Bernard Amegadzie Markers and Methods for Assessing and Treating Psoriasis and Related Disorders
US12054724B2 (en) * 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
JP2022504683A (ja) * 2018-10-11 2022-01-13 デシベル セラピューティクス インコーポレイテッド 耳適応症の治療のためのａａｖ１ベクター及びその使用
WO2020097372A1 (en) * 2018-11-07 2020-05-14 Akouos, Inc. Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear
KR20220133854A (ko) * 2019-09-30 2022-10-05 어플라이드 제네틱스 테크놀로지스 코퍼레이션 유전적 청력 상실 치료를 위한 아데노 관련 바이러스(aav) 시스템

Also Published As

Publication number Publication date
KR20230069157A (ko) 2023-05-18
EP4211151A1 (en) 2023-07-19
WO2022056444A1 (en) 2022-03-17
BR112023004605A2 (pt) 2023-04-11
CA3191533A1 (en) 2022-03-17
IL301057A (en) 2023-05-01
JP2023541443A (ja) 2023-10-02
TW202227476A (zh) 2022-07-16
AU2021339843A1 (en) 2023-04-06

Similar Documents

Publication Publication Date Title
MX2023002978A (es) Virus adenoasociado recombinante (raav) que codifica la union gap beta 2 (gjb2) y sus usos.
ES2940433T3 (es) Expresión transgénica y procesamiento potenciados
US9896665B2 (en) Proviral plasmids and production of recombinant adeno-associated virus
McIntosh et al. Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine
US12018285B2 (en) Integration sites in CHO cells
Johnen et al. Sleeping Beauty transposon-mediated transfection of retinal and iris pigment epithelial cells
Gu et al. Transduction of adeno-associated virus vectors targeting hair cells and supporting cells in the neonatal mouse cochlea
Nair et al. Effect of different UCOE-promoter combinations in creation of engineered cell lines for the production of Factor VIII
RU2017142006A (ru) Векторы на основе аденоассоциированных вирусов для лечения мукополисахаридозов
EA027693B1 (ru) Рекомбинантный репликационно-компетентный ретровирус с цитозиндезаминазной активностью для генной терапии нарушений клеточной пролиферации
Eggenschwiler et al. Sustained knockdown of a disease-causing gene in patient-specific induced pluripotent stem cells using lentiviral vector-based gene therapy
Weltner et al. Induced pluripotent stem cell clones reprogrammed via recombinant adeno-associated virus-mediated transduction contain integrated vector sequences
US20230257767A1 (en) Promoter of Hspa5 Gene
do Amaral et al. Approaches for recombinant human factor IX production in serum-free suspension cultures
Duran Alonso et al. Transcription factor induced conversion of human fibroblasts towards the hair cell lineage
EP4273236A1 (en) Hek293t cell strain having high dispersibility and screening method therefor
MX2023012518A (es) Sistemas de producción estables para la producción de virus adenoasociado.
Park et al. Amplification of EBNA-1 through a single-plasmid vector-based gene amplification system in HEK293 cells as an efficient transient gene expression system
KR20200055141A (ko) 바이러스 생성 페이로드에 대해 독성이 낮은 변형된 ec7 세포(modified ec7 cells having low toxicity to viral production payloads)
EP4417689A1 (en) Hek293 cell line adapted to serum-free suspension culture and use thereof
WO2023221530A1 (zh) 一种治疗听力损伤的双载体系统及其应用
Min et al. Hybrid cell line development system utilizing site-specific integration and methotrexate-mediated gene amplification in Chinese hamster ovary cells
US20240150793A1 (en) Vector, linear covalently closed dna production method using vector, parvovirus vector production method, and parvovirus-vector-producing cell production method
US20230313187A1 (en) Modifications of mammalian cells using artificial micro-rna to alter their properties and the compositions of their products
Kamada et al. Establishment of ultra long‐lived cell lines by transfection of TERT into normal human fibroblast TIG‐1 and their characterization