IL234007A0 - Systemic delivery and regulated expression of paracrine genes for cardiovascular disease and other conditions - Google Patents

Systemic delivery and regulated expression of paracrine genes for cardiovascular disease and other conditions

Info

Publication number
IL234007A0
IL234007A0 IL234007A IL23400714A IL234007A0 IL 234007 A0 IL234007 A0 IL 234007A0 IL 234007 A IL234007 A IL 234007A IL 23400714 A IL23400714 A IL 23400714A IL 234007 A0 IL234007 A0 IL 234007A0
Authority
IL
Israel
Prior art keywords
paracrine
genes
conditions
cardiovascular diseases
systemic delivery
Prior art date
Application number
IL234007A
Other languages
English (en)
Hebrew (he)
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 IL234007A0 publication Critical patent/IL234007A0/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
    • A61K38/2228—Corticotropin releasing factor [CRF] (Urotensin)
    • 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
    • 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
    • A61K38/2242—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)
    • 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)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (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)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Endocrinology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Hematology (AREA)
  • Cardiology (AREA)
  • Cell Biology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Communicable Diseases (AREA)
IL234007A 2012-02-14 2014-08-07 Systemic delivery and regulated expression of paracrine genes for cardiovascular disease and other conditions IL234007A0 (en)

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
IL234007A0 true IL234007A0 (en) 2014-09-30

Family

ID=48984885

Family Applications (3)

Application Number Title Priority Date Filing Date
IL234007A IL234007A0 (en) 2012-02-14 2014-08-07 Systemic delivery and regulated expression of paracrine genes for cardiovascular disease and other conditions
IL261172A IL261172A (en) 2012-02-14 2018-08-15 Systemic delivery and regulated expression of paracrine genes for cardiovascular disease and other conditions
IL26260018A IL262600B (en) 2012-02-14 2018-10-25 Vectors and their medical uses

Family Applications After (2)

Application Number Title Priority Date Filing Date
IL261172A IL261172A (en) 2012-02-14 2018-08-15 Systemic delivery and regulated expression of paracrine genes for cardiovascular disease and other conditions
IL26260018A IL262600B (en) 2012-02-14 2018-10-25 Vectors and their medical uses

Country Status (18)

Country Link
US (3) US20150118287A1 (cg-RX-API-DMAC7.html)
EP (2) EP3311830A1 (cg-RX-API-DMAC7.html)
JP (3) JP6562632B2 (cg-RX-API-DMAC7.html)
KR (1) KR102107482B1 (cg-RX-API-DMAC7.html)
CN (2) CN108310396B (cg-RX-API-DMAC7.html)
AU (2) AU2013221615B2 (cg-RX-API-DMAC7.html)
BR (1) BR112014020127A2 (cg-RX-API-DMAC7.html)
CA (1) CA2864100A1 (cg-RX-API-DMAC7.html)
DK (1) DK2814513T3 (cg-RX-API-DMAC7.html)
ES (1) ES2660492T3 (cg-RX-API-DMAC7.html)
HK (1) HK1254639A1 (cg-RX-API-DMAC7.html)
HU (1) HUE036640T2 (cg-RX-API-DMAC7.html)
IL (3) IL234007A0 (cg-RX-API-DMAC7.html)
IN (1) IN2014DN06789A (cg-RX-API-DMAC7.html)
PL (1) PL2814513T3 (cg-RX-API-DMAC7.html)
PT (1) PT2814513T (cg-RX-API-DMAC7.html)
RU (1) RU2642605C2 (cg-RX-API-DMAC7.html)
WO (1) WO2013123094A2 (cg-RX-API-DMAC7.html)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
RU2642605C2 (ru) * 2012-02-14 2018-01-25 Те Риджентс Оф Те Юниверсити Оф Калифорния Системная доставка и регулируемая экспрессия паракринных генов для лечения сердечно-сосудистых и иных заболеваний
CN106456803A (zh) * 2014-04-03 2017-02-22 加利福尼亚大学董事会 编码尿皮质素‑2和相关基因的病毒载体的全身递送以治疗糖尿病相关性心脏功能障碍和充血性心力衰竭
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3209311B1 (en) 2014-10-21 2024-03-06 University of Massachusetts Recombinant aav variants and uses thereof
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
AU2016260190A1 (en) * 2015-05-12 2017-11-23 The Regents Of The University Of California Peptide treatment for inflammation and fibrosis
HK1250623A1 (zh) * 2015-05-28 2019-01-11 Cornell University 腺相关病毒介导的c1ei递送作为用於血管性水肿的疗法
US11299751B2 (en) 2016-04-29 2022-04-12 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
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
IL302748B1 (en) 2016-05-18 2025-09-01 Voyager Therapeutics Inc Modulatory polynucleotides
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
EP3526333A4 (en) 2016-10-13 2020-07-29 University of Massachusetts AAV CAPSIDE DESIGNS
CA3050910A1 (en) * 2017-01-23 2018-07-26 Rutgers, The State University Of New Jersey Compositions for reducing sarcolipin expression and preventing and treating muscular dystrophy and cardiomyopathy and methods of use
RU2740713C1 (ru) 2017-02-28 2021-01-20 Генемедицине Ко., Лтд. Противораковая композиция, содержащая опухолеспецифический онколитический аденовирус и ингибитор контрольной точки иммунного ответа
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
EP3618839A4 (en) 2017-05-05 2021-06-09 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
EP3654860A1 (en) 2017-07-17 2020-05-27 Voyager Therapeutics, Inc. Trajectory array guide system
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
WO2019067840A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. RESTRICTING CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE FROM FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CN111479924B (zh) 2017-10-16 2024-06-14 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
ES2986944T3 (es) * 2018-06-11 2024-11-13 Univ California Vector vírico que codifica la proteína ELOVL2 para el tratamiento de la degeneración macular relacionada con la edad
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2021251589A1 (ko) * 2020-06-12 2021-12-16 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
CA2239775C (en) 1995-12-18 2008-07-15 Collagen Corporation Crosslinked polymer compositions and methods for their use
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
JP2001503755A (ja) * 1996-11-01 2001-03-21 ユーロジーン・リミテッド Ｎｏまたはプロスタサイクリン産生を刺激する薬剤の治療的使用およびデリバリー装置
US6630137B1 (en) 1997-04-28 2003-10-07 Eli Lilly And Company Activated protein C formulations
EA005157B1 (ru) * 1997-05-06 2004-12-30 Те Риджентс Оф Те Юниверсити Оф Калифорния Методы и составы для лечения сердечной недостаточности и вентрикулярной коррекции путем доставки in vivo ангиогенных трансгенов
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
ES2313784T3 (es) * 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
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
HK1048593A1 (zh) * 1999-11-05 2003-04-11 The Regents Of The University Of California 用於通过体内基因输送治疗心血管疾病的技术和组合物
AU2460001A (en) * 1999-12-27 2001-07-09 Regents Of The University Of California, The Gene therapy for congestive heart failure
CA2405291A1 (en) 2000-04-06 2001-10-18 Wayne P. Franco Methods of using growth factors for treating heart disease
US7442390B2 (en) 2000-06-05 2008-10-28 University Of South Florida Method for enhancing engraftment of cells using mesenchymal progenitor cells
CA2418144C (en) * 2000-08-04 2013-06-18 Research Development 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
EP1559777A1 (en) 2001-04-24 2005-08-03 Hokkaido Technology Licensing Office Co., Ltd. Process for maturating small heptocyte-rich colonies into liver tissue
JP4810062B2 (ja) * 2001-12-17 2011-11-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）血清型８の配列
US7192923B2 (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
EP1773298A1 (en) 2004-08-06 2007-04-18 Biospectrum, Inc. Multiple layered liposome and preparation method thereof
MXPA06014403A (es) 2004-08-20 2007-05-08 Artes Medical Usa Inc Metodos de administracion de microparticulas combinadas con componentes autologos del cuerpo.
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
JP4959570B2 (ja) 2004-10-18 2012-06-27 日東電工株式会社 細胞内ペプチド送達
KR20070112155A (ko) * 2005-02-08 2007-11-22 리서치 디벨럽먼트 파운데이션 가용성 Ｇ-단백질 연결 수용체(ｓＧＰＣＲ)와 관련된조성물 및 방법
US7759120B2 (en) 2005-03-02 2010-07-20 Kps Bay Medical, Inc. Seeding implantable medical devices with cells
US20070059288A1 (en) * 2005-03-31 2007-03-15 Dinsmore Jonathan H Treatment for heart disease
EP1937213B1 (en) 2005-07-27 2017-10-25 Protiva Biotherapeutics Inc. Systems and methods for manufacturing liposomes
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
EP2114437A2 (en) * 2006-10-16 2009-11-11 ConjuChem Biotechnologies Inc. Modified corticotropin releasing factor peptides and uses thereof
AU2007200990B8 (en) 2006-11-21 2011-05-12 Living Cell Technologies Limited Cell Implantation To Prevent and/or Treat Hearing Loss
JP2011503207A (ja) * 2007-11-16 2011-01-27 サン ディエゴ ステート ユニバーシティ リサーチ ファウンデーション 循環系細胞におけるｐｉｍ−１活性を操作するための組成物および方法
EP2225002A4 (en) * 2007-12-31 2011-06-22 Nanocor Therapeutics Inc RNA INTERFERENCE FOR THE TREATMENT OF CARDIAC SUFFICIENCIES
EP3150219A1 (en) * 2008-05-16 2017-04-05 Corthera, Inc. Method of treating chronic heart failure
ES2546287T3 (es) * 2008-05-16 2015-09-22 Corthera, Inc. Relaxina para uso en el tratamiento de la disnea asociada con la insuficiencia cardiaca aguda
US10040838B2 (en) * 2008-11-04 2018-08-07 Janssen Pharmaceutica Nv CRHR2 peptide agonists and uses thereof
US8734809B2 (en) * 2009-05-28 2014-05-27 University Of Massachusetts 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
RU2642605C2 (ru) * 2012-02-14 2018-01-25 Те Риджентс Оф Те Юниверсити Оф Калифорния Системная доставка и регулируемая экспрессия паракринных генов для лечения сердечно-сосудистых и иных заболеваний
US11065347B2 (en) * 2016-01-19 2021-07-20 The Regents Of The University Of California Methods for the treatment of Danon disease and other disorders of autophagy

Also Published As

Publication number Publication date
ES2660492T3 (es) 2018-03-22
US20180010148A1 (en) 2018-01-11
IN2014DN06789A (cg-RX-API-DMAC7.html) 2015-05-22
AU2013221615B2 (en) 2018-02-22
CN104220098A (zh) 2014-12-17
CN104220098B (zh) 2018-04-10
US20150118287A1 (en) 2015-04-30
HK1203845A1 (en) 2015-11-06
IL262600B (en) 2019-11-28
KR20140124403A (ko) 2014-10-24
IL262600A (en) 2018-12-31
AU2018200719B2 (en) 2019-10-24
AU2018200719A1 (en) 2018-02-22
BR112014020127A2 (pt) 2020-06-30
WO2013123094A2 (en) 2013-08-22
RU2014137169A (ru) 2016-04-10
US10918738B2 (en) 2021-02-16
CN108310396B (zh) 2022-07-15
PT2814513T (pt) 2018-03-02
JP2019135263A (ja) 2019-08-15
JP2015508760A (ja) 2015-03-23
HK1254639A1 (en) 2019-07-26
PL2814513T3 (pl) 2018-06-29
KR102107482B1 (ko) 2020-05-08
US20190367945A1 (en) 2019-12-05
RU2642605C2 (ru) 2018-01-25
DK2814513T3 (en) 2018-02-26
HUE036640T2 (hu) 2018-07-30
CN108310396A (zh) 2018-07-24
EP2814513B1 (en) 2017-12-20
EP3311830A1 (en) 2018-04-25
EP2814513A4 (en) 2016-01-13
EP2814513A2 (en) 2014-12-24
US10202618B2 (en) 2019-02-12
JP6562632B2 (ja) 2019-08-21
JP2017203048A (ja) 2017-11-16
IL261172A (en) 2018-10-31
CA2864100A1 (en) 2013-08-22
AU2013221615A1 (en) 2014-08-28

Similar Documents

Publication Publication Date Title
IL261172A (en) Systemic delivery and regulated expression of paracrine genes for cardiovascular disease and other conditions
IL233946A0 (en) A classified method for the administration of nucleic acids and preparations and its uses
PL2899182T3 (pl) Aryloalkilowe estry 4-aminopikolinianów oraz ich zastosowanie jako herbicydy
IL238638A0 (en) Dosage and Administration of Oligonucleotide Cancer Therapies
IL237639A0 (en) Alkanedial and alkanedial derivatives at the 17th carbon position of oleinolic acid and methods of using them
IL235450A0 (en) Formulations and methods for administering antiprogestins through the vagina
EP3007705A4 (en) Systemic in vivo delivery of oligonucleotides
PT2815769T (pt) Fam19a5 para utilização no diagnóstico e tratamento de lesões do snc
IL235831A0 (en) A new dosage form and formulation of aveditrol
IL237359A0 (en) Transgenic algae and their use for oral administration of proteins
EP2877482A4 (en) PROCESS AND INTERMEDIATES USED IN THE PREPARATION OF ABIRATERONE ACETATE
ZA201504442B (en) Regulation of gene expression
SG11201502954TA (en) Rnai pharmaceutical composition for suppressing expression of kras gene
ZA201505049B (en) Transmucosal delivery of glatiramer acetate
ZA201500739B (en) Isolating traffic-enhancing mutants of drug delivery protein
ZA201308905B (en) Nasal pharmaceutical formulation comprising fluticasone
AP2015008545A0 (en) Products of ethanol products
SG11201406942SA (en) Trans-2-decenoic acid derivative and drug containing same
IL233445A0 (en) Methods and preparations for gene transfer
EP2697649A4 (en) GENE EXPRESSION PROFILE FOR THERAPEUTIC RESPONSE TO VEGF HEMMER
GB201109291D0 (en) Muco-adhesive tablet for systemic administration of insulin
AU2012905531A0 (en) Delivery of Surface Care Products