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AR076445A1 - Regeneracion de islotes pancreaticos y reversion de la diabetes por medio de la administracion in vivo de genes del factor de transcripcion de los islotes. composicion. metodo. vector. celula. - Google Patents

Regeneracion de islotes pancreaticos y reversion de la diabetes por medio de la administracion in vivo de genes del factor de transcripcion de los islotes. composicion. metodo. vector. celula.

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Publication number
AR076445A1
AR076445A1 ARP090104420A ARP090104420A AR076445A1 AR 076445 A1 AR076445 A1 AR 076445A1 AR P090104420 A ARP090104420 A AR P090104420A AR P090104420 A ARP090104420 A AR P090104420A AR 076445 A1 AR076445 A1 AR 076445A1
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AR
Argentina
Prior art keywords
insulin
composition
exon
promoter
pancreas
Prior art date
Application number
ARP090104420A
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English (en)
Inventor
Paul A Grayburn
Shuyuan Chen
Jiahuan Ding
Original Assignee
Baylor Res Inst
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Publication date
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Publication of AR076445A1 publication Critical patent/AR076445A1/es

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K41/00Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
    • A61K41/0028Disruption, e.g. by heat or ultrasounds, sonophysical or sonochemical activation, e.g. thermosensitive or heat-sensitive liposomes, disruption of calculi with a medicinal preparation and ultrasounds
    • CCHEMISTRY; METALLURGY
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083Medicinal 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 administration regime
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • A61P5/48Drugs for disorders of the endocrine system of the pancreatic hormones
    • A61P5/50Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10Cells modified by introduction of foreign genetic material

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Biophysics (AREA)
  • Hematology (AREA)
  • Plant Pathology (AREA)
  • Obesity (AREA)
  • Emergency Medicine (AREA)
  • Dispersion Chemistry (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Reivindicacion 1: Una composicion para la destruccion de microburbujas dirigida por ultrasonido en el páncreas, caracterizada porque comprende un complejo preensamblado de liposomas y ácidos nucleicos en microburbujas, donde el complejo preensamblado de liposomas y ácidos nucleicos comprende un gen NeuroD bajo el control de un promotor de insulina, que comprende uno o más genes reguladores que responden a la insulina, unidos operativamente a una region promotora de la insulina que comprende un fragmento genomico del promotor de la insulina que comprende una region no traducida 5', el exon 1, el intron 1 y el exon 2 del gen de la insulina, donde la destruccion de la microburbuja en un sitio blanco en el páncreas permite introducir el ácido nucleico en las células del páncreas en las que ha tenido lugar dicha destruccion dirigida por ultrasonido, donde las células en las que se ha incorporado el ácido nucleico expresan insulina en respuesta a los niveles elevados de glucosa en la sangre. Reivindicacion 3: La composicion de la reivindicacion 1, caracterizada porque también comprende uno o más genes seleccionados entre uno o más genes reguladores que responden a la insulina, unidos operativamente a una region promotora de la insulina seleccionada entre ngn3, GLP1, PDX1, Mafa, la betacelulina, Nkx2.2, Nkx6.1, PAX4, lsI1, la ciclina D2 (y otros miembros de la familia de la ciclina), CDK4 (y otros miembros de la familia de la quinasa dependiente de ciclina) y ARNpi contra inhibidores de la quinasa dependientes de ciclina, tales como p16 y otros miembros de la familia INK4 o p27 y otros miembros de la familia CIP/KIP. Reivindicacion 4: La composicion de la reivindicacion 1, caracterizada porque también comprende un agente que se co-administra con la composicion, donde el agente se selecciona entre un agente anti-apoptotico, un agente antiinflamatorio, un inhibidor de JNK, un GLP-1, un tacrolimus, un sirolimus, una anakinra, una poliamida de Dervin o combinaciones de éstos. Reivindicacion 5: Una composicion para regenerar células beta pancreáticas por medio de la destruccion de microburbujas dirigida por ultrasonido en el páncreas, caracterizada porque comprende microburbujas que comprenden NeuroD, donde las microburbujas comprenden lípidos que liberan NeuroD mediante la destruccion dirigida por ultrasonido en el páncreas. Reivindicacion 7: La composicion de la reivindicacion 5, caracterizada porque NeuroD comprende un gen NeuroD bajo el control de un promotor CUBI, RIP2.1, RIP3.1 o HIP3.1, y donde NeuroD se expresa en las células donde se desea la expresion por medio de la destruccion de microburbujas dirigida por ultrasonido. Reivindicacion 25: Un ácido nucleico aislado caracterizado porque comprende una region promotora de la insulina que comprende un fragmento genomico del promotor de la insulina que comprende una region no traducida 5', el exon 1, el intron 1 y el exon 2 del gen de la insulina, hacia el extremo 5' de uno o más genes que responden a al insulina. Reivindicacion 32: La composicion de la reivindicacion 26, caracterizada porque el complejo preensamblado de liposomas y ácidos nucleicos comprende 1,2- dipalmitoil-sn-glicero-3-fosfatidilcolina y 1,2-dipalmitoil-sn-glicero-3-fosfatidiletanolamina glicerol en combinacion con un plásmido. Reivindicacion 35: Un vector caracterizado porque comprende un gen de hexoquinasa bajo el control de un promotor que comprende uno o más genes reguladores que responden a la insulina, unidos operativamente a una region promotora de la insulina que comprende un fragmento genomico del promotor de la insulina que comprende una region no traducida 5', el exon 1, el intron 1 y el exon 2 del gen de la insulina. Reivindicacion 41: Una célula caracterizada porque se le ha conferido la capacidad de responder a la insulina con un método que comprende inyectar en una célula un complejo preensamblado de liposomas y ácidos nucleicos en una microburbuja, donde el complejo preensamblado de liposomas y ácidos nucleicos comprende un gen NeuroD bajo el control de un promotor de insulina que comprende uno o más genes reguladores que responden a la insulina, unidos operativamente a una region promotora de la insulina que comprende un fragmento genomico del promotor de la insulina que comprende una region no traducida 5', el exon 1, el intron 1 y el exon 2 del gen de la insulina, donde la destruccion de la microburbuja en un sitio blanco en el páncreas permite introducir el ácido nucleico en las células del páncreas en las que ha tenido lugar dicha destruccion dirigida por ultrasonido, donde las células en las que se ha incorporado el ácido nucleico expresan insulina en respuesta a los niveles elevados de glucosa en la sangre.
ARP090104420A 2008-11-13 2009-11-13 Regeneracion de islotes pancreaticos y reversion de la diabetes por medio de la administracion in vivo de genes del factor de transcripcion de los islotes. composicion. metodo. vector. celula. AR076445A1 (es)

Applications Claiming Priority (1)

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US11440708P 2008-11-13 2008-11-13

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AR076445A1 true AR076445A1 (es) 2011-06-15

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ARP090104420A AR076445A1 (es) 2008-11-13 2009-11-13 Regeneracion de islotes pancreaticos y reversion de la diabetes por medio de la administracion in vivo de genes del factor de transcripcion de los islotes. composicion. metodo. vector. celula.

Country Status (14)

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US (2) US20110287086A1 (es)
EP (1) EP2350297A4 (es)
JP (2) JP2012508585A (es)
KR (1) KR101305931B1 (es)
CN (1) CN102282263B (es)
AR (1) AR076445A1 (es)
AU (1) AU2009313875B2 (es)
BR (1) BRPI0922030A2 (es)
CA (1) CA2743668A1 (es)
IL (1) IL212881A0 (es)
MX (1) MX2011005047A (es)
NZ (3) NZ592821A (es)
TW (1) TW201029669A (es)
WO (1) WO2010057045A2 (es)

Families Citing this family (9)

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US20120093914A1 (en) 2008-11-24 2012-04-19 Moma Therapeutics Implantable liposome embedded matrix composition, uses thereof, and polycaprolactone particles as scaffolds for tissue regeneration
WO2011094352A1 (en) * 2010-01-27 2011-08-04 Baylor Research Institute In-vivo non-viral gene delivery of human vascular endothelial growth factor following islet transplantation
AR080806A1 (es) * 2010-03-24 2012-05-09 Baylor Res Inst Expresion del gen neurod1 en celulas epiteliales pancreaticas no endocrinas (nepec)
US8916534B2 (en) 2010-10-08 2014-12-23 Mina Therapeutics Limited Methods of inducing insulin production
GB201205158D0 (en) 2012-03-23 2012-05-09 Univ Leeds Apparatus and method for manipulating entrained particles
EP3065706A4 (en) * 2013-11-08 2017-11-29 Baylor Research Institute Nuclear localization of glp-1 stimulates myocardial regeneration and reverses heart failure
EP3594348B1 (en) 2013-11-22 2021-09-08 MiNA Therapeutics Limited C/ebp alpha short activating rna compositions and methods of use
JP7594298B2 (ja) * 2018-08-01 2024-12-04 オハイオ・ステイト・イノベーション・ファウンデーション 皮膚をインスリン産生組織にリプログラミングするための組成物および方法
CN113122538A (zh) * 2021-04-15 2021-07-16 遵义医科大学附属医院 一种靶向敲减Rip3基因表达的shRNA、重组载体及其应用

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US6911324B2 (en) * 2001-10-18 2005-06-28 The Regents Of The University Of California Induction of beta cell differentiation in human cells
US7141240B2 (en) * 2002-03-12 2006-11-28 Cedars-Sinai Medical Center Glucose-dependent insulin-secreting cells transfected with a nucleotide sequence encoding GLP-1
US20040132679A1 (en) * 2002-09-03 2004-07-08 Baylor College Of Medicine Induction of pancreatic islet formation
WO2007008220A2 (en) * 2004-08-05 2007-01-18 Baylor Research Institute Gene or drug delivery system
EP1951881A4 (en) * 2005-11-07 2009-03-11 Gen Hospital Corp METHOD AND COMPOSITIONS FOR MODULATING THE AGING OF STEM CELLS
CN101573445A (zh) * 2006-09-22 2009-11-04 贝勒研究院 由胰腺腺泡细胞向胰岛素生成细胞的体内转化

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EP2350297A4 (en) 2012-05-09
WO2010057045A3 (en) 2010-09-16
CA2743668A1 (en) 2010-05-20
JP2014168463A (ja) 2014-09-18
WO2010057045A2 (en) 2010-05-20
KR20110086594A (ko) 2011-07-28
WO2010057045A8 (en) 2011-02-03
AU2009313875A1 (en) 2010-05-20
NZ592821A (en) 2012-06-29
AU2009313875B2 (en) 2013-01-10
US20110287086A1 (en) 2011-11-24
NZ595273A (en) 2012-10-26
EP2350297A2 (en) 2011-08-03
BRPI0922030A2 (pt) 2018-10-16
CN102282263A (zh) 2011-12-14
KR101305931B1 (ko) 2013-09-12
CN102282263B (zh) 2015-02-11
NZ602474A (en) 2013-02-22
TW201029669A (en) 2010-08-16
JP2012508585A (ja) 2012-04-12
MX2011005047A (es) 2011-07-29
JP5813161B2 (ja) 2015-11-17
US20140294924A1 (en) 2014-10-02
IL212881A0 (en) 2011-07-31

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