CA2836921C - Hepatocyte based insulin gene therapy for diabetes - Google Patents

Hepatocyte based insulin gene therapy for diabetes Download PDF

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Publication number
CA2836921C
CA2836921C CA2836921A CA2836921A CA2836921C CA 2836921 C CA2836921 C CA 2836921C CA 2836921 A CA2836921 A CA 2836921A CA 2836921 A CA2836921 A CA 2836921A CA 2836921 C CA2836921 C CA 2836921C
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Canada
Prior art keywords
vector
insulin
seq
residues
enhancer
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CA2836921A
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English (en)
French (fr)
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CA2836921A1 (en
Inventor
Tausif Alam
Hans Sollinger
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Wisconsin Alumni Research Foundation
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Wisconsin Alumni Research Foundation
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    • 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
    • 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
    • C07K14/575—Hormones
    • C07K14/62—Insulins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/37—Digestive system
    • A61K35/407—Liver; Hepatocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • 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
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2840/105—Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Diabetes (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Endocrinology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Physiology (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • Nutrition Science (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Emergency Medicine (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
CA2836921A 2011-06-07 2012-06-06 Hepatocyte based insulin gene therapy for diabetes Active CA2836921C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161494134P 2011-06-07 2011-06-07
US61/494,134 2011-06-07
PCT/US2012/041099 WO2012170531A1 (en) 2011-06-07 2012-06-06 Hepatocyte based insulin gene therapy for diabetes

Publications (2)

Publication Number Publication Date
CA2836921A1 CA2836921A1 (en) 2012-12-13
CA2836921C true CA2836921C (en) 2020-08-18

Family

ID=46246296

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2836921A Active CA2836921C (en) 2011-06-07 2012-06-06 Hepatocyte based insulin gene therapy for diabetes

Country Status (6)

Country Link
US (6) US20120315255A1 (enExample)
EP (2) EP2718319B1 (enExample)
CA (1) CA2836921C (enExample)
ES (2) ES2774491T3 (enExample)
NO (1) NO2718319T3 (enExample)
WO (1) WO2012170531A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NO2718319T3 (enExample) * 2011-06-07 2018-05-05
WO2013143698A1 (en) * 2012-03-27 2013-10-03 Curevac Gmbh Artificial nucleic acid molecules
US9897565B1 (en) 2012-09-11 2018-02-20 Aseko, Inc. System and method for optimizing insulin dosages for diabetic subjects
US9171343B1 (en) 2012-09-11 2015-10-27 Aseko, Inc. Means and method for improved glycemic control for diabetic patients
US9898585B2 (en) 2014-01-31 2018-02-20 Aseko, Inc. Method and system for insulin management
US9486580B2 (en) 2014-01-31 2016-11-08 Aseko, Inc. Insulin management
US11081226B2 (en) 2014-10-27 2021-08-03 Aseko, Inc. Method and controller for administering recommended insulin dosages to a patient
JP6989262B2 (ja) 2014-10-27 2022-01-05 アセコー インコーポレイテッド 皮下外来患者管理
EP3337402B1 (en) 2015-08-20 2026-01-07 Glytec, LLC Diabetes management therapy advisor
WO2020025357A1 (en) * 2018-08-02 2020-02-06 Novozymes A/S Preparation of combinatorial libraries of dna constructs using introns
CN120349985A (zh) * 2018-10-12 2025-07-22 建新公司 通过肝导向基因替代疗法治疗严重pku的改良的人pah的产生
CN113939595A (zh) 2019-06-07 2022-01-14 瑞泽恩制药公司 包括人源化白蛋白基因座的非人动物
TW202302858A (zh) * 2021-03-16 2023-01-16 威斯康辛校友研究基金會 治療糖尿病之胰島素基因療法
CA3252672A1 (en) * 2022-05-09 2023-11-16 Endsulin, Inc. VARIANT OF PREPROINSULIN AND CONSTRUCTIONS FOR INSULIN EXPRESSION AND THE TREATMENT OF DIABETES

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5427940A (en) 1991-06-03 1995-06-27 Board Of Regents, The University Of Texas System Engineered cells producing insulin in response to glucose
SG47470A1 (en) 1991-04-25 1998-04-17 Univ Brown Res Found Implantable biocompatible immunoisolatory vehicle for delivery of a selected therapeutic products
US5534404A (en) 1993-12-10 1996-07-09 Cytotherapeutics, Inc. Glucose responsive insulin secreting β-cell lines and method for producing same
US7425443B2 (en) 1997-01-21 2008-09-16 Wisconsin Alumni Research Foundation Treatment of diabetes with synthetic beta cells
US6352857B1 (en) 1997-01-21 2002-03-05 Wisconsin Alumni Research Foundation Treatment of diabetes with synthetic beta cells
US8021875B2 (en) * 2001-08-27 2011-09-20 Roche Madison Inc. Methods for expression of transgenes
WO2007127408A2 (en) 2006-04-28 2007-11-08 Tulane University Health Sciences Center Methods for treating diabetes
DE102008042352A1 (de) 2008-09-25 2010-04-08 Robert Bosch Gmbh Ansteuerung eines Synchrongleichrichters
NO2718319T3 (enExample) * 2011-06-07 2018-05-05

Also Published As

Publication number Publication date
NO2718319T3 (enExample) 2018-05-05
WO2012170531A1 (en) 2012-12-13
US20140170123A1 (en) 2014-06-19
ES2774491T3 (es) 2020-07-21
US20260117250A1 (en) 2026-04-30
EP2718319A1 (en) 2014-04-16
US10443072B2 (en) 2019-10-15
US20190382791A1 (en) 2019-12-19
ES2658487T3 (es) 2018-03-12
EP2718319B1 (en) 2017-12-06
US12163147B2 (en) 2024-12-10
US20120315255A1 (en) 2012-12-13
EP3293197A1 (en) 2018-03-14
EP3293197B1 (en) 2020-01-08
US9732356B2 (en) 2017-08-15
CA2836921A1 (en) 2012-12-13
US20210388381A1 (en) 2021-12-16
US20170342441A1 (en) 2017-11-30

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