WO2005037226A3 - Genetically engineered enteroendocrine cells for treating glucose-related metabolic disorders - Google Patents

Genetically engineered enteroendocrine cells for treating glucose-related metabolic disorders Download PDF

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Publication number
WO2005037226A3
WO2005037226A3 PCT/US2004/034377 US2004034377W WO2005037226A3 WO 2005037226 A3 WO2005037226 A3 WO 2005037226A3 US 2004034377 W US2004034377 W US 2004034377W WO 2005037226 A3 WO2005037226 A3 WO 2005037226A3
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WIPO (PCT)
Prior art keywords
cells
vector
selection
transduced
genetically engineered
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Application number
PCT/US2004/034377
Other languages
French (fr)
Other versions
WO2005037226A2 (en
Inventor
Shiue-Cheng Tang
Athanassios Sambanis
Original Assignee
Georgia Tech Res Inst
Shiue-Cheng Tang
Athanassios Sambanis
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 Georgia Tech Res Inst, Shiue-Cheng Tang, Athanassios Sambanis filed Critical Georgia Tech Res Inst
Publication of WO2005037226A2 publication Critical patent/WO2005037226A2/en
Publication of WO2005037226A3 publication Critical patent/WO2005037226A3/en

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    • 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
    • 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
    • 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

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  • 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)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Methods and compositions for restoration of regulated insulin release in response to blood glucose levels in an individual in need thereof are disclosed. A recombinant adeno-associated virus expression vector expressing preproinsulin or insulin is used for selective transduction of enteroendocrine cells. Cells can be transfected with vectors where the genes of interest are under the control of tissue-specific promoters. Other appropriate enhancers or transcriptional regulators may be included in the vector to modulate expression. Cells can be transduced in vivo or manipulated ex vivo and transplanted into an individual in need of treatment. A selection gene or reporter gene can optionally be included in the vector for selection of transduced cells after expression of the vector. Genes encoding luminescent markers can optionally be included in the vector to allow for selection of transduced cells using fluorescence-based selection procedures.
PCT/US2004/034377 2003-10-17 2004-10-18 Genetically engineered enteroendocrine cells for treating glucose-related metabolic disorders WO2005037226A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US51204403P 2003-10-17 2003-10-17
US60/512,044 2003-10-17

Publications (2)

Publication Number Publication Date
WO2005037226A2 WO2005037226A2 (en) 2005-04-28
WO2005037226A3 true WO2005037226A3 (en) 2005-07-21

Family

ID=34465307

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2004/034377 WO2005037226A2 (en) 2003-10-17 2004-10-18 Genetically engineered enteroendocrine cells for treating glucose-related metabolic disorders

Country Status (1)

Country Link
WO (1) WO2005037226A2 (en)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5572972B2 (en) 2009-03-16 2014-08-20 Ｊｎｃ株式会社 Screening method for drugs such as insulin secretagogues
PT2506857T (en) 2009-12-01 2018-05-14 Translate Bio Inc Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
EP2569430B1 (en) 2010-05-12 2018-10-17 The Trustees of Columbia University in the City of New York Methods for producing enteroendocrine cells that make and secrete insulin
US20140112895A1 (en) * 2010-10-08 2014-04-24 Universiti Putra Malaysia Glp-1 promoter mediated insulin expression for the treatment of diabetes
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
TR201910686T4 (en) 2011-06-08 2019-08-21 Translate Bio Inc Lipid nanoparticle compositions and methods for Mrna delivery.
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
PL2968586T3 (en) 2013-03-14 2019-01-31 Translate Bio, Inc. Cftr mrna compositions and related methods and uses
EA201591229A1 (en) 2013-03-14 2016-01-29 Шир Хьюман Дженетик Терапис, Инк. METHODS OF CLEANING MATRIX RNA
EP4276176A3 (en) 2013-10-22 2024-01-10 Translate Bio, Inc. Mrna therapy for argininosuccinate synthetase deficiency
EP3060258A1 (en) 2013-10-22 2016-08-31 Shire Human Genetic Therapies, Inc. Mrna therapy for phenylketonuria
EA201691696A1 (en) 2014-04-25 2017-03-31 Шир Хьюман Дженетик Терапис, Инк. METHODS OF CLEANING MATRIX RNA
EP3160503B1 (en) 2014-06-26 2021-02-17 The Trustees of Columbia University in the City of New York Inhibition of serotonin expression in gut enteroendocrine cells results in conversion to insulin-positive cells
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
KR102415896B1 (en) 2015-06-23 2022-06-30 더 칠드런스 호스피탈 오브 필라델피아 Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
MA47603A (en) 2017-02-27 2020-01-01 Translate Bio Inc NEW ARNM CFTR WITH OPTIMIZED CODONS
WO2018213476A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
WO2019087988A1 (en) * 2017-10-30 2019-05-09 公立大学法人横浜市立大学 Construct having structure and cell mass linked together
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
WO2022072324A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Isl1 and lhx3 vector

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
PATERNA J.C. ET AL: "Influence of promoter and WHV post-transcriptional regulatory element on AAV-mediated transgene expression in the rat brain", GENE THERAPY, vol. 7, 2000, pages 1304 - 1311, XP002953221 *
PENG L. ET AL: "Construction of Recombinant Adeno-Associated Virus Vector Containing the Rat Preproinsulin II Gene", JOURNAL OF SURGICAL RESEARCH, vol. 69, 1997, pages 193 - 198, XP002986085 *
TANG ET AL: "Development of Geneticaly Engineered Human Intestinal Cells for Regulated Insulin Secretion Using rAAV-mediated Gene Transfer", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 303, no. 2, April 2003 (2003-04-01), pages 645 - 652, XP002986086 *

Also Published As

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