[go: up one dir, main page]

AR078848A1 - Tecnologia terapeutica de arn de interferencia dirigido al oncogen pdx -1 en tumores neuroendocrinos donde se expresa pdx-1 - Google Patents

Tecnologia terapeutica de arn de interferencia dirigido al oncogen pdx -1 en tumores neuroendocrinos donde se expresa pdx-1

Info

Publication number
AR078848A1
AR078848A1 ARP100104018A AR078848A1 AR 078848 A1 AR078848 A1 AR 078848A1 AR P100104018 A ARP100104018 A AR P100104018A AR 078848 A1 AR078848 A1 AR 078848A1
Authority
AR
Argentina
Prior art keywords
pdx
oncogene
mrna
expression
promoter
Prior art date
Application number
Other languages
English (en)
Inventor
F Charles Brunicardi
John J Nemunaitis
Donald Rao
Original Assignee
Gradalis Inc
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 Gradalis Inc filed Critical Gradalis Inc
Publication of AR078848A1 publication Critical patent/AR078848A1/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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1135—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against oncogenes or tumor suppressor genes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Oncology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

Composicion bifuncional basada en ARNph y métodos para anular la expresion del oncogen PDX-1 en células blanco. Métodos para administrar vectores de expresion que contienen ARNph a tejidos blanco donde se expresa el oncogen PDX-1. Reivindicacion 1: Un vector de expresion caracterizado porque comprende: un promotor; y un ácido nucleico insertado unido operativamente al promotor, donde el inserto codifica uno o más ARN pequenos en horquilla (ARNph) que son capaces de hibridar con una region de un transcripto de ARNm que codifica un oncogen PDX-1 y que inhiben la expresion del oncogen PDX-1 a través de la interferencia a nivel del ARN. Reivindicacion 7: Un sistema de administracion terapéutica caracterizado porque comprende: un agente que es un vehículo terapéutico y un vector de expresion que comprende un promotor y un ácido nucleico insertado unido operativamente al promotor, que codifica uno o más ARN pequenos en horquilla (ARNph) que hibridan con una region de un transcripto de ARNm que codifica un oncogen PDX-1 y que inhiben la expresion del oncogen PDX-1 a través de la interferencia a nivel del ARN. Reivindicacion 8: El sistema de administracion de la reivindicacion 7, caracterizado porque el agente que es un vehículo terapéutico es una nanopartícula de ADN compactada. Reivindicacion 11: El sistema de administracion de la reivindicacion 8, caracterizado porque las nanopartículas de ADN compactadas también están encapsuladas en un liposoma.
ARP100104018 2009-10-30 2010-10-29 Tecnologia terapeutica de arn de interferencia dirigido al oncogen pdx -1 en tumores neuroendocrinos donde se expresa pdx-1 AR078848A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US25686709P 2009-10-30 2009-10-30
US12/913,515 US8361983B2 (en) 2009-10-30 2010-10-27 Therapeutic RNA interference technology targeted to the PDX-1 oncogene in PDX-1 expressing neuroendocrine tumors
PCT/US2010/054350 WO2011053660A2 (en) 2009-10-30 2010-10-27 Novel therapeutic rna interference technology targeted to the pdx-1 oncogene in pdx-1 expressing neuroendocrine tumors

Publications (1)

Publication Number Publication Date
AR078848A1 true AR078848A1 (es) 2011-12-07

Family

ID=43922974

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP100104018 AR078848A1 (es) 2009-10-30 2010-10-29 Tecnologia terapeutica de arn de interferencia dirigido al oncogen pdx -1 en tumores neuroendocrinos donde se expresa pdx-1

Country Status (11)

Country Link
US (2) US8361983B2 (es)
EP (1) EP2507374B1 (es)
JP (2) JP6007103B2 (es)
KR (1) KR101543350B1 (es)
CN (1) CN102782139B (es)
AR (1) AR078848A1 (es)
AU (1) AU2010313441B2 (es)
CA (1) CA2777539C (es)
IL (2) IL219312A0 (es)
TW (2) TWI418369B (es)
WO (1) WO2011053660A2 (es)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8603991B2 (en) 2005-11-18 2013-12-10 Gradalis, Inc. Individualized cancer therapy
AU2010313441B2 (en) * 2009-10-30 2013-05-02 Baylor College Of Medicine Novel therapeutic RNA interference technology targeted to the PDX-1 oncogene in PDX-1 expressing neuroendocrine tumors
US9132146B2 (en) 2009-12-23 2015-09-15 Gradalis, Inc. Furin-knockdown and GM-CSF-augmented (FANG) cancer vaccine
SG181881A1 (en) 2009-12-23 2012-07-30 Gradalis Inc Furin-knockdown bi-functional rna
WO2013036879A1 (en) * 2011-09-08 2013-03-14 Gradalis, Inc. Compositions and methods for treating prostate cancer
US20130266639A1 (en) * 2012-03-28 2013-10-10 Baylor College Of Medicine METHODS FOR TREATING TRIPLE NEGATIVE BREAST CANCER USING BIFUNCTIONAL SRC 3 shRNA
WO2013148824A1 (en) * 2012-03-28 2013-10-03 Gradalis, Inc. METHODS AND COMPOSITIONS TO TREAT CANCER USING BIFUNCTIONAL SRC-3 shRNA
US20130259927A1 (en) * 2012-04-02 2013-10-03 Gradalis, Inc. Ewing's Sarcoma Bifunctional shRNA Design
AU2013259387B2 (en) 2012-05-09 2016-12-15 Strike Bio, Inc. Bi-functional short-hairpin rna (bi-shrna) specific for single-nucleotide kras mutations
US8825022B2 (en) 2012-09-14 2014-09-02 International Business Machines Corporation Information sharing for third party applications in cellular telecommunication infrastructures
CN115335085A (zh) 2020-01-13 2022-11-11 格兰达利斯有限公司 用编码gm-csf的多核苷酸和额外药剂治疗癌症的方法

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5858973A (en) * 1994-02-23 1999-01-12 The General Hospital Corporation Transcription factor and uses therefor
US6716824B1 (en) 1999-10-22 2004-04-06 F. Charles Brunicardi Treatment of pancreatic adenocarcinoma by cytotoxic gene therapy
CA2410265A1 (en) * 2000-05-31 2001-12-06 Copernicus Therapeutics, Inc. Lyophilizable and enhanced compacted nucleic acids
US20020042388A1 (en) * 2001-05-01 2002-04-11 Cooper Mark J. Lyophilizable and enhanced compacted nucleic acids
US7037520B2 (en) 2002-03-22 2006-05-02 Baylor College Of Medicine Reversible masking of liposomal complexes for targeted delivery
US8252526B2 (en) * 2006-11-09 2012-08-28 Gradalis, Inc. ShRNA molecules and methods of use thereof
WO2008101087A1 (en) * 2007-02-14 2008-08-21 Ontherex Llc Compositions and methods for modulation of pdx-1
CN101348512B (zh) * 2007-07-20 2012-08-15 郑州威瑞生物技术有限公司 一种抗肿瘤的腺病毒制剂
EP2195429A2 (en) * 2007-08-20 2010-06-16 Senesco Technologies, Inc. Use of eif-5a1 sirna to protect islets cells from apoptosis and to preserve their functionality
CA2758023A1 (en) * 2008-04-09 2009-10-15 Cornell University Commensal bacteria as signal mediators within a mammalian host
AU2010313441B2 (en) * 2009-10-30 2013-05-02 Baylor College Of Medicine Novel therapeutic RNA interference technology targeted to the PDX-1 oncogene in PDX-1 expressing neuroendocrine tumors

Also Published As

Publication number Publication date
IL238977A0 (en) 2015-07-30
JP2013509183A (ja) 2013-03-14
US8361983B2 (en) 2013-01-29
JP6007103B2 (ja) 2016-10-12
US20130084331A1 (en) 2013-04-04
EP2507374A2 (en) 2012-10-10
TW201125594A (en) 2011-08-01
US20110117183A1 (en) 2011-05-19
EP2507374A4 (en) 2013-10-23
TWI533894B (zh) 2016-05-21
CN102782139A (zh) 2012-11-14
TWI418369B (zh) 2013-12-11
CA2777539A1 (en) 2011-05-05
CN102782139B (zh) 2015-11-25
IL238977A (en) 2016-10-31
AU2010313441B2 (en) 2013-05-02
KR101543350B1 (ko) 2015-08-11
AU2010313441A1 (en) 2012-05-03
US9133459B2 (en) 2015-09-15
EP2507374B1 (en) 2016-12-07
IL219312A0 (en) 2012-06-28
CA2777539C (en) 2016-09-13
HK1176964A1 (zh) 2013-08-09
WO2011053660A2 (en) 2011-05-05
KR20120072387A (ko) 2012-07-03
TW201406405A (zh) 2014-02-16
WO2011053660A3 (en) 2011-10-06
JP2014207907A (ja) 2014-11-06

Similar Documents

Publication Publication Date Title
AR078848A1 (es) Tecnologia terapeutica de arn de interferencia dirigido al oncogen pdx -1 en tumores neuroendocrinos donde se expresa pdx-1
Joo et al. Porous silicon–graphene oxide core–shell nanoparticles for targeted delivery of siRNA to the injured brain
Ling Non-coding RNAs: therapeutic strategies and delivery systems
CN107106592B (zh) 用于p21基因调节的rna干扰剂
Lee et al. Stability and cellular uptake of polymerized siRNA (poly-siRNA)/polyethylenimine (PEI) complexes for efficient gene silencing
Yin et al. Overcoming chemoresistance in cancer via combined microRNA therapeutics with anticancer drugs using multifunctional magnetic core–shell nanoparticles
Kang et al. Liver-targeted siRNA delivery by polyethylenimine (PEI)-pullulan carrier
Liu et al. Targeted delivery of Dicer-substrate siRNAs using a dual targeting peptide decorated dendrimer delivery system
PE20230179A1 (es) Metodos y composiciones para el tratamiento de un trastorno asociado con angiotensinogeno (agt)
JP2019006792A (ja) 治療剤の送達方法
JP2013046616A5 (es)
JP2008005844A (ja) 特定遺伝子の発現を抑制するための方法および薬剤
WO2018183808A9 (en) Antiviral therapeutic
US20220143197A1 (en) siRNA Inhibition Of Human Antigen R Expression For Treatment of Cancer
WO2006108718B1 (en) Micro rna
IN2014DN10390A (es)
JP2018533956A5 (es)
KR20220144831A (ko) 암호화 리보핵산의 기관 보호적 발현 및 조절을 위한 조성물 및 방법
Kim et al. Efficient Delivery of Globotriaosylceramide Synthase siRNA using Polyhistidine‐Incorporated Lipid Nanoparticles
Liu et al. RETRACTED: Downregulation of hsa_circ_0001836 Induces Pyroptosis Cell Death in Glioma Cells via Epigenetically Upregulating NLRP1
TW201628629A (zh) 用於生物體內及體外遞送核酸系藥物之新穎糖醇系組成物
Kobelt et al. A brief introduction to current cancer gene therapy
AR091327A1 (es) DISEÑO DE ARNhc BIFUNCIONAL EN EL SARCOMA DE EWING
FR3058061A1 (fr) Nouvelle utilisation d'oligonucleotides double brin
US20240384245A1 (en) Method for treating a disease

Legal Events

Date Code Title Description
FB Suspension of granting procedure