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MX2021004391A - Proteinas inteinas y usos de las mismas. - Google Patents

Proteinas inteinas y usos de las mismas.

Info

Publication number
MX2021004391A
MX2021004391A MX2021004391A MX2021004391A MX2021004391A MX 2021004391 A MX2021004391 A MX 2021004391A MX 2021004391 A MX2021004391 A MX 2021004391A MX 2021004391 A MX2021004391 A MX 2021004391A MX 2021004391 A MX2021004391 A MX 2021004391A
Authority
MX
Mexico
Prior art keywords
intein proteins
intein
proteins
constructs
vectors
Prior art date
Application number
MX2021004391A
Other languages
English (en)
Inventor
Ivana Trapani
Alberto Auricchio
Patrizia Tornabene
Original Assignee
Fond Telethon
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 Fond Telethon filed Critical Fond Telethon
Publication of MX2021004391A publication Critical patent/MX2021004391A/es

Links

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
    • 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
    • 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
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Hematology (AREA)
  • Diabetes (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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

La presente invención se refiere a constructos, vectores, células hospederas relativas y composiciones farmacéuticas que permiten una terapia génica eficaz, en particular de genes mayores de 5 kb.
MX2021004391A 2018-10-15 2019-10-15 Proteinas inteinas y usos de las mismas. MX2021004391A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18200490 2018-10-15
EP19169116 2019-04-12
PCT/EP2019/078020 WO2020079034A2 (en) 2018-10-15 2019-10-15 Intein proteins and uses thereof

Publications (1)

Publication Number Publication Date
MX2021004391A true MX2021004391A (es) 2021-08-16

Family

ID=68234008

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021004391A MX2021004391A (es) 2018-10-15 2019-10-15 Proteinas inteinas y usos de las mismas.

Country Status (12)

Country Link
US (1) US20210371878A1 (es)
EP (1) EP3867387A2 (es)
JP (2) JP2022512718A (es)
KR (1) KR20210104661A (es)
CN (1) CN113348249B (es)
AU (1) AU2019360372A1 (es)
BR (1) BR112021007221A2 (es)
CA (1) CA3116606A1 (es)
IL (2) IL320368A (es)
MX (1) MX2021004391A (es)
SG (1) SG11202103886XA (es)
WO (1) WO2020079034A2 (es)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10494645B2 (en) 2013-04-18 2019-12-03 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
WO2019005192A1 (en) 2017-06-28 2019-01-03 Corning Research & Development Corporation COMPACT MULTIPLE CONNECTING FIBER OPTICAL FIBER CONNECTORS, AND CABLE ASSEMBLIES AND METHODS OF MAKING SAME
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
WO2020112354A1 (en) 2018-11-29 2020-06-04 Corning Research & Development Corporation Multiports having connection ports with rotating actuators and method for making the same
EP4045957B1 (en) 2019-10-18 2023-12-13 Corning Research & Development Corporation Terminals having optical connection ports with securing features providing stable retention forces
GB201918693D0 (en) * 2019-12-18 2020-01-29 Univ Southampton Peptide
EP4100518A4 (en) * 2020-02-07 2024-06-05 The Children's Medical Center Corporation Large gene vectors and delivery and uses thereof
EP3885440A1 (en) * 2020-03-26 2021-09-29 Splicebio, S.L. Split inteins and their uses
WO2021209574A1 (en) 2020-04-15 2021-10-21 Fondazione Telethon Constructs comprising inteins
WO2022056440A1 (en) * 2020-09-14 2022-03-17 President And Fellows Of Harvard College Dual-aav vector delivery of pcdh15 and uses thereof
CA3197116A1 (en) 2020-10-30 2022-05-05 Stephen Paul CAPPANNARI Female fiber optic connectors having a rocker latch arm and methods of making the same
US11880076B2 (en) 2020-11-30 2024-01-23 Corning Research & Development Corporation Fiber optic adapter assemblies including a conversion housing and a release housing
US20250099616A1 (en) * 2021-07-23 2025-03-27 University Of Washington Generation of large proteins by co-delivery of multiple vectors
EP4424714A4 (en) * 2021-10-29 2025-10-15 Shanghai Sinobay Biotechnology Co Ltd Condition-Controlled Splicable Chimeric Antigen Receptor Molecule and Its Use
CN114854694A (zh) * 2022-04-29 2022-08-05 四川轻化工大学 一种高通量筛选新冠药物的荧光素酶互补体系及其构建方法与应用
EP4612166A1 (en) * 2022-11-01 2025-09-10 Memorial Sloan-Kettering Cancer Center Intein-based sorting system and modular chimeric polypeptides
IT202300007968A1 (it) 2023-04-21 2024-10-21 Fond Telethon Ets Metodi di editing genomico e costrutti
WO2024238891A1 (en) * 2023-05-18 2024-11-21 The Regents Of The University Of California Gene therapy for docks deficiency
WO2024251077A1 (zh) * 2023-06-07 2024-12-12 北京中因科技有限公司 表达盒组合及其应用
WO2024258925A1 (en) 2023-06-12 2024-12-19 Children's Hospital Medical Center Aav-cftr vectors and methods of using same
CN116925239B (zh) * 2023-07-17 2024-10-18 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
WO2025051758A1 (en) 2023-09-04 2025-03-13 Splicebio, Sl Use of split intein for the treatment of scn1a associated disease
WO2025153705A1 (en) 2024-01-18 2025-07-24 Splicebio, Sl Use of split intein for the treatment of myo7a-associated disease
WO2026013288A1 (en) * 2024-07-12 2026-01-15 Splicebio, Sl Split intein for use in the treatment of cep290-associated disease

Family Cites Families (19)

* Cited by examiner, † Cited by third party
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US3919208A (en) 1973-10-09 1975-11-11 Yeda Res & Dev 7-(Cyanomethylaryl)acetamide-cephalosporin derivatives
US5166331A (en) 1983-10-10 1992-11-24 Fidia, S.P.A. Hyaluronics acid fractions, methods for the preparation thereof, and pharmaceutical compositions containing same
WO2001029243A1 (en) * 1999-10-15 2001-04-26 Dalhousie University Method and vector for producing and transferring trans-spliced peptides
WO2009132455A1 (en) 2008-04-30 2009-11-05 Paul Xiang-Qin Liu Protein splicing using short terminal split inteins
US20100098772A1 (en) 2008-10-21 2010-04-22 Allergan, Inc. Drug delivery systems and methods for treating neovascularization
EP2665744B1 (en) * 2011-01-20 2015-04-08 University Of Rochester Macrocyclic compounds with a hybrid peptidic/non-peptidic backbone and methods for their preparation
WO2012125445A2 (en) 2011-03-11 2012-09-20 President And Fellows Of Harvard College Small molecule-dependent inteins and uses thereof
ES2618632T3 (es) * 2011-09-28 2017-06-21 Zera Intein Protein Solutions, S.L. Inteínas divididas y usos de éstas
KR102166277B1 (ko) 2013-04-12 2020-10-15 삼성전자주식회사 무선 통신 네트워크를 이용한 운전 지원 방법 및 장치와 그 시스템
US10494645B2 (en) * 2013-04-18 2019-12-03 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
NL2013235B1 (en) 2014-07-22 2016-08-16 Douwe Egberts Bv Pad for use in a machine for preparing at least one part of a single beverage serving, system including a machine and method for preparing at least one part of a single beverage serving with such a system.
WO2016069774A1 (en) * 2014-10-28 2016-05-06 Agrivida, Inc. Methods and compositions for stabilizing trans-splicing intein modified proteases
US10066027B2 (en) * 2015-01-09 2018-09-04 Ohio State Innovation Foundation Protein production systems and methods thereof
CA2979120A1 (en) * 2015-03-03 2016-09-09 Fondazione Telethon Multiple aav vector system for expressing genes over 5 kilobases, comprising at least one degradation signal and uses thereof
CA3051195A1 (en) * 2016-01-29 2017-08-03 The Trustees Of Princeton University Split inteins with exceptional splicing activity
CA2968112C (en) 2016-05-26 2025-09-23 Op-Hygiene Ip Gmbh MAINTENANCE OF A DISPENSER INTENDED FOR AN INSTALLATION WITH MULTIPLE BATHROOMS
MX2018015629A (es) * 2016-06-15 2019-09-26 Univ Oxford Innovation Ltd Sistema de vector viral adeno-asociado traslapado doble para la expresion de abc4a.
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
UY37636A (es) * 2017-03-21 2018-10-31 Univ Muenchen Ludwig Maximilians Terapia génica para el tratamiento de retinitis pigmentosa ligada a cngb1

Also Published As

Publication number Publication date
JP2025020111A (ja) 2025-02-12
SG11202103886XA (en) 2021-05-28
IL282362B1 (en) 2025-06-01
JP2022512718A (ja) 2022-02-07
CN113348249A (zh) 2021-09-03
IL282362A (en) 2021-06-30
EP3867387A2 (en) 2021-08-25
WO2020079034A3 (en) 2020-06-18
US20210371878A1 (en) 2021-12-02
IL320368A (en) 2025-06-01
CA3116606A1 (en) 2020-04-23
AU2019360372A1 (en) 2021-06-03
IL282362B2 (en) 2025-10-01
KR20210104661A (ko) 2021-08-25
BR112021007221A2 (pt) 2021-08-10
CN113348249B (zh) 2025-09-23
WO2020079034A2 (en) 2020-04-23

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