IN2014DN06789A - - Google Patents

Info

Publication number
IN2014DN06789A
IN2014DN06789A IN6789DEN2014A IN2014DN06789A IN 2014DN06789 A IN2014DN06789 A IN 2014DN06789A IN 6789DEN2014 A IN6789DEN2014 A IN 6789DEN2014A IN 2014DN06789 A IN2014DN06789 A IN 2014DN06789A
Authority
IN
India
Prior art keywords
paracrine
nucleic acid
encoding nucleic
gene
individual
Prior art date
Application number
Other languages
English (en)
Inventor
H Kirk Hammond
Mei Hua Gao
Original Assignee
Univ California
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 Univ California filed Critical Univ California
Publication of IN2014DN06789A publication Critical patent/IN2014DN06789A/en

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
    • C12N15/861—Adenoviral vectors
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
    • 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
    • A61K31/65—Tetracyclines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/2221—Relaxins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/2228—Corticotropin releasing factor [CRF] (Urotensin)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/2242—Atrial natriuretic factor complex: Atriopeptins, atrial natriuretic protein [ANP]; Cardionatrin, Cardiodilatin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/25—Growth hormone-releasing factor [GH-RF], i.e. somatoliberin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/52—Isomerases (5)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6901—Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or 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
    • 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
    • A61K48/005—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/06—Ointments; Bases therefor; Other semi-solid forms, e.g. creams, sticks, gels
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • 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
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A61P3/04—Anorexiants; Antiobesity agents
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/075—Adenoviridae
    • 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
    • C07K14/57509—Corticotropin releasing factor [CRF] (Urotensin)
    • 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
    • C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00—Preparations for use in therapy
    • 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
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—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
    • 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
    • C12N2830/001—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
    • C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Endocrinology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Hematology (AREA)
  • Cardiology (AREA)
  • Cell Biology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Obesity (AREA)
IN6789DEN2014 2012-02-14 2013-02-13 IN2014DN06789A (en, 2012)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261598772P 2012-02-14 2012-02-14
PCT/US2013/025997 WO2013123094A2 (en) 2012-02-14 2013-02-13 Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions

Publications (1)

Publication Number Publication Date
IN2014DN06789A true IN2014DN06789A (en, 2012) 2015-05-22

Family

ID=48984885

Family Applications (1)

Application Number Title Priority Date Filing Date
IN6789DEN2014 IN2014DN06789A (en, 2012) 2012-02-14 2013-02-13

Country Status (18)

Country Link
US (3) US20150118287A1 (en, 2012)
EP (2) EP3311830A1 (en, 2012)
JP (3) JP6562632B2 (en, 2012)
KR (1) KR102107482B1 (en, 2012)
CN (2) CN104220098B (en, 2012)
AU (2) AU2013221615B2 (en, 2012)
BR (1) BR112014020127A2 (en, 2012)
CA (1) CA2864100A1 (en, 2012)
DK (1) DK2814513T3 (en, 2012)
ES (1) ES2660492T3 (en, 2012)
HK (1) HK1254639A1 (en, 2012)
HU (1) HUE036640T2 (en, 2012)
IL (3) IL234007A0 (en, 2012)
IN (1) IN2014DN06789A (en, 2012)
PL (1) PL2814513T3 (en, 2012)
PT (1) PT2814513T (en, 2012)
RU (1) RU2642605C2 (en, 2012)
WO (1) WO2013123094A2 (en, 2012)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP3311830A1 (en) * 2012-02-14 2018-04-25 The Regents of The University of California Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
EP3125948A4 (en) * 2014-04-03 2017-11-15 The Regents of the University of California Systemic delivery of virus vectors encoding urocortin-2 and related genes to treat diabetes-related cardiac dysfunction and congestive heart failure
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
AU2015335923B2 (en) * 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
JP6401871B2 (ja) 2014-11-05 2018-10-10 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． パーキンソン病の治療のためのａａｄｃポリヌクレオチド
KR20230169197A (ko) 2014-11-14 2023-12-15 보이저 테라퓨틱스, 인크. 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법
EP3218386B1 (en) 2014-11-14 2021-03-17 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
JP7372026B2 (ja) * 2015-05-12 2023-10-31 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 炎症および線維症のペプチド治療
HK1250623A1 (zh) * 2015-05-28 2019-01-11 Cornell University 腺相關病毒介導的c1ei遞送作為用於血管性水腫的療法
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
CN110475573A (zh) * 2017-01-23 2019-11-19 新泽西鲁特格斯州立大学 用于减少肌脂蛋白表达以及预防和治疗肌营养不良症和心肌病的组合物及其使用方法
CN119236118A (zh) 2017-02-28 2025-01-03 基因药物株式会社 含肿瘤特异性溶瘤腺病毒和免疫检查点抑制剂的抗癌组合物
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
US11760987B2 (en) 2017-09-07 2023-09-19 The Regents Of The University Of California Compositions and methods for the treatment or prevention of heart failure
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
JP7502991B2 (ja) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症（ａｌｓ）の治療
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
BR112020023082A2 (pt) 2018-05-15 2021-02-09 Voyager Therapeutics, Inc. composições e métodos para o tratamento de doença de parkinson
US10632211B2 (en) * 2018-06-11 2020-04-28 The Regents Of The University Of California Demethylation to treat eye disease
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
KR102418528B1 (ko) * 2020-06-12 2022-07-11 주식회사 젠셀메드 다중 표적화 재조합 헤르페스 심플렉스 바이러스 및 그 용도
CN112826823B (zh) * 2021-01-11 2022-03-08 中国人民解放军军事科学院军事医学研究院 多西环素在制备治疗和/或预防和/或缓解和/或改善心肌病药物中的应用

Family Cites Families (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5217879A (en) 1989-01-12 1993-06-08 Washington University Infectious Sindbis virus vectors
US5733542A (en) 1990-11-16 1998-03-31 Haynesworth; Stephen E. Enhancing bone marrow engraftment using MSCS
US5270192A (en) 1991-02-07 1993-12-14 Monsanto Company Biological artificial liver
US5846743A (en) 1995-02-22 1998-12-08 Brigham And Women's Hospital, Inc. Polyphoshoinositide binding peptides for intracellular drug delivery
US7361332B2 (en) 1995-03-17 2008-04-22 The Regents Of The University Of California Treating tumors using implants comprising combinations of allogeneic cells
US6214797B1 (en) * 1995-06-13 2001-04-10 The Salk Institute For Biological Studies Urocortin peptides, nucleic acid encoding same methods for using same
US5721118A (en) 1995-10-31 1998-02-24 The Regents Of The University Of California, San Diego Mammalian artificial chromosomes and methods of using same
PT1704878E (pt) 1995-12-18 2013-07-17 Angiodevice Internat Gmbh Composições de polímeros reticulados e métodos para a sua utilização
US6025155A (en) 1996-04-10 2000-02-15 Chromos Molecular Systems, Inc. Artificial chromosomes, uses thereof and methods for preparing artificial chromosomes
US6933331B2 (en) 1998-05-22 2005-08-23 Nanoproducts Corporation Nanotechnology for drug delivery, contrast agents and biomedical implants
US5874268A (en) 1996-09-23 1999-02-23 Duke University Method of introducing exogenous compounds into cells by electroporation and apparatus for same
EP1488761A1 (en) * 1996-11-01 2004-12-22 Ark Therapeutics Limited Device for delivering a therapeutic agent to a blood vessel
US6630137B1 (en) 1997-04-28 2003-10-07 Eli Lilly And Company Activated protein C formulations
CN1267331A (zh) * 1997-05-06 2000-09-20 加利福尼亚大学董事会 通过在体内运送血管生成转基因来治疗心力衰竭和心室重建的技术和组合物
US20050095227A1 (en) * 1997-07-22 2005-05-05 The General Hospital Corporation Treating heart failure
SE9704076D0 (sv) 1997-11-06 1997-11-06 Holdingbolaget Vid Goeteborgs Method for permeabilisation of cell structures and use thereof
US6886568B2 (en) 1998-04-08 2005-05-03 The Johns Hopkins University Method for fabricating cell-containing implants
JP4060531B2 (ja) * 1998-05-28 2008-03-12 アメリカ合衆国 Ａａｖ５ベクターおよびその使用
US6589503B1 (en) 1998-06-20 2003-07-08 Washington University Membrane-permeant peptide complexes for medical imaging, diagnostics, and pharmaceutical therapy
US6958212B1 (en) 1999-02-01 2005-10-25 Eidgenossische Technische Hochschule Zurich Conjugate addition reactions for the controlled delivery of pharmaceutically active compounds
CN1433325A (zh) * 1999-11-05 2003-07-30 加利福尼亚大学董事会 通过体内基因递送治疗心血管疾病的技术和组合物
WO2001048164A2 (en) * 1999-12-27 2001-07-05 The Regents Of The University Of California Modified adenylylcyclase type vi useful in gene therapy for congestive heart failure
EP1604024A4 (en) 2000-04-06 2008-04-23 Wayne P Franco METHODS OF USING GROWTH FACTORS FOR TREATING CARDIOPATHY
US7442390B2 (en) 2000-06-05 2008-10-28 University Of South Florida Method for enhancing engraftment of cells using mesenchymal progenitor cells
AU8468301A (en) * 2000-08-04 2002-02-18 Res Dev Foundation Urocortin proteins and uses thereof
NZ523803A (en) * 2000-08-04 2004-08-27 Res Dev Foundation Human urocortin-related polypeptide (URP) and uses thereof to rreat a pathophysiological state, eg stress, anxiety and appetite dysfunction
JPWO2002088332A1 (ja) 2001-04-24 2004-08-19 北海道ティー・エル・オー株式会社 小型肝細胞高含有コロニー、その調製方法、その肝組織への成熟化方法、成熟化した小型肝細胞高含有コロニーを用いた薬物機能の推定方法
EP2359869B1 (en) * 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor
US7192924B2 (en) * 2002-01-16 2007-03-20 The Procter & Gamble Company Corticotropin releasing factor 2 receptor agonists
US7166133B2 (en) 2002-06-13 2007-01-23 Kensey Nash Corporation Devices and methods for treating defects in the tissue of a living being
US20040151766A1 (en) 2003-01-30 2004-08-05 Monahan Sean D. Protein and peptide delivery to mammalian cells in vitro
US7241455B2 (en) 2003-04-08 2007-07-10 Boston Scientific Scimed, Inc. Implantable or insertable medical devices containing radiation-crosslinked polymer for controlled delivery of a therapeutic agent
WO2005011520A2 (en) 2003-07-25 2005-02-10 Dexcom, Inc. Oxygen enhancing membrane systems for implantable devices
US7794706B2 (en) 2003-10-14 2010-09-14 Medivas, Llc Bioactive wound dressings and implantable devices and methods of use
US20050136121A1 (en) 2003-12-22 2005-06-23 Shear/Kershman Laboratories, Inc. Oral peptide delivery system with improved bioavailability
CA2549966A1 (en) 2003-12-24 2005-07-07 Ltt Bio-Pharma Co., Ltd. Drug-containing nanoparticle, process for producing the same and parenterally administered preparation from the nanoparticle
US20050244463A1 (en) 2004-04-30 2005-11-03 Allergan, Inc. Sustained release intraocular implants and methods for treating ocular vasculopathies
US8658203B2 (en) 2004-05-03 2014-02-25 Merrimack Pharmaceuticals, Inc. Liposomes useful for drug delivery to the brain
WO2006014035A1 (en) 2004-08-06 2006-02-09 Biospectrum, Inc. Multiple layered liposome and preparation method thereof
CA2569736A1 (en) 2004-08-20 2006-03-02 Russell Anderson Methods of administering microparticles combined with autologous body components
WO2006026325A2 (en) 2004-08-26 2006-03-09 Pathak Chandrashekhar P Implantable tissue compositions and method
US7351423B2 (en) 2004-09-01 2008-04-01 Depuy Spine, Inc. Musculo-skeletal implant having a bioactive gradient
US7501486B2 (en) 2004-09-07 2009-03-10 Burnham Institute For Medical Research Peptides that selectively home to heart vasculature and related conjugates and methods
EP1812074B1 (en) 2004-10-18 2010-12-29 Nitto Denko Corporation Intracellular peptide delivery
JP4859248B2 (ja) * 2005-02-08 2012-01-25 リサーチ ディベロップメント ファウンデーション 可溶性Ｇタンパク質共役型レセプター（ｓＧＰＣＲ）に関する組成物および方法
US7759120B2 (en) 2005-03-02 2010-07-20 Kps Bay Medical, Inc. Seeding implantable medical devices with cells
JP2008537942A (ja) * 2005-03-31 2008-10-02 マイトジェン， インコーポレイテッド 心疾患のための処置
JP5639338B2 (ja) 2005-07-27 2014-12-10 プロチバ バイオセラピューティクス インコーポレイティッド リポソームの製造システムおよび製造方法
US20070265221A1 (en) 2006-05-09 2007-11-15 Weiss Robert G Methods to improve creatine kinase metabolism and contractile function in cardiac muscle for the treatment of heart failure
US7982018B2 (en) * 2006-10-16 2011-07-19 Conjuchem, Llc Modified corticotropin releasing factor peptides and uses thereof
US20080119909A1 (en) 2006-11-21 2008-05-22 Neurotrophincell Pty Limited Cell implantation to prevent and/or treat hearing loss
EP2209889B1 (en) * 2007-11-16 2016-07-20 San Diego State University Research Foundation Compositions for manipulating pim-1 activity in cardiac cells
JP2011512326A (ja) * 2007-12-31 2011-04-21 ナノコア セラピューティクス，インコーポレイテッド 心不全の治療用のｒｎａ干渉
SG191587A1 (en) * 2008-05-16 2013-07-31 Corthera Inc Treating dyspnea associated with acute heart failure with relaxin
MX336145B (es) * 2008-05-16 2016-01-08 Corthera Inc Metodo para el tratamiento de insuficiencia cardiaca cronica.
SG171957A1 (en) * 2008-11-04 2011-07-28 Janssen Pharmaceutica Nv Crhr2 peptide agonists and uses thereof
WO2010138263A2 (en) * 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP2496248A2 (en) * 2009-11-04 2012-09-12 Janssen Pharmaceutica, N.V. Method for treating heart failure with stresscopin-like peptides
EP3311830A1 (en) * 2012-02-14 2018-04-25 The Regents of The University of California Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
CA3011731A1 (en) * 2016-01-19 2017-07-27 Eric D. Adler Methods for the treatment of danon disease and other disorders of autophagy

Also Published As

Publication number Publication date
CA2864100A1 (en) 2013-08-22
IL261172A (en) 2018-10-31
HK1254639A1 (en) 2019-07-26
RU2014137169A (ru) 2016-04-10
JP2019135263A (ja) 2019-08-15
DK2814513T3 (en) 2018-02-26
US20180010148A1 (en) 2018-01-11
CN104220098A (zh) 2014-12-17
IL262600B (en) 2019-11-28
AU2013221615B2 (en) 2018-02-22
US10918738B2 (en) 2021-02-16
PT2814513T (pt) 2018-03-02
CN104220098B (zh) 2018-04-10
CN108310396A (zh) 2018-07-24
EP2814513A4 (en) 2016-01-13
BR112014020127A2 (pt) 2020-06-30
HK1203845A1 (en) 2015-11-06
CN108310396B (zh) 2022-07-15
KR102107482B1 (ko) 2020-05-08
EP2814513B1 (en) 2017-12-20
RU2642605C2 (ru) 2018-01-25
IL262600A (en) 2018-12-31
US10202618B2 (en) 2019-02-12
US20150118287A1 (en) 2015-04-30
WO2013123094A2 (en) 2013-08-22
AU2018200719B2 (en) 2019-10-24
JP6562632B2 (ja) 2019-08-21
EP3311830A1 (en) 2018-04-25
ES2660492T3 (es) 2018-03-22
EP2814513A2 (en) 2014-12-24
JP2017203048A (ja) 2017-11-16
HUE036640T2 (hu) 2018-07-30
AU2013221615A1 (en) 2014-08-28
JP2015508760A (ja) 2015-03-23
US20190367945A1 (en) 2019-12-05
KR20140124403A (ko) 2014-10-24
AU2018200719A1 (en) 2018-02-22
PL2814513T3 (pl) 2018-06-29
IL234007A0 (en) 2014-09-30

Similar Documents

Publication Publication Date Title
IN2014DN06789A (en, 2012)
NZ704275A (en) Aav-mediated gene therapy for rpgr x-linked retinal degeneration
MX2016002152A (es) Metodo para aumentar la expresion de proteinas codificadas por arn.
MX2015009963A (es) Metodos y composición farmacéutica para el tratamiento y la prevención de cardiomiopatía debido a falta de energía.
BR112016016290A2 (pt) Composições farmacêuticas de insulina de ação rápida, conjunto de infusão, sistema de conjunto de infusão e usos de análogos monoméricos ou diméricos de insulina
BR112016028023A2 (pt) Composições e métodos de administração de tratamentos para infecções virais latentes
MA40057A (fr) Traitement thérapeutique de maladie cutanée au moyen de micro-organismes cutanés commensaux recombinants
MX357803B (es) Moléculas de ácido nucleico artificiales.
MX2015014464A (es) Vectores para terapia genica para tratar cardiomiopatía.
MX367842B (es) Método compartimentado de administración de ácidos nucleicos y composiciones y usos del mismo.
NZ587105A (en) Tissue protective peptides and peptide analogs for preventing and treating diseases and disorders associated with tissue damage
MX2013005341A (es) Formulaciones liquidas de anticuerpos anti-tnf-alfa de alta concentracion mejoradas.
IN2014DN09554A (en, 2012)
BR112015021884A2 (pt) vetores compreendendo sequências de polinucleotídeos de stuffer/agente de enchimento e métodos de uso
IN2014DN06920A (en, 2012)
SG10201804552WA (en) Nanoparticle compositions, formulations thereof, and uses therefor
BR112012029975A2 (pt) peptídeo como medicamento, em particular para o tratamento do câncer.
CY1123793T1 (el) Γονιδιακη θεραπεια rpgr για μελαγχρωστικη αμφιβληστροειδοπαθεια
MX391136B (es) FRAGMENTOS DE OspA ESTABILIZADOS CON CISTEÍNA Y MÉTODO DE USO
PH12014501388A1 (en) A recombinant koi herpesvirus (khv) and vaccine for the prevention of a disease caused by khv
WO2012083302A3 (en) Compositions and methods for the treatment or prevention of human adenovirus-36 infection
MX2015011905A (es) Uso de levocetirizina y montelukast en el tratamiento de la vasculitis.
MX366134B (es) Angiotensina en el tratamiento de condiciones cerebrales.
EA201491702A1 (ru) Оптимизированные терапевтические агенты для подкожного введения
MY168778A (en) Oligonucleotide chelate complex-polypeptide compositions and methods