WO2004032973A3 - Use of sns insulator of sea urchin for the gene therapy of erythroid cell diseases - Google Patents

Use of sns insulator of sea urchin for the gene therapy of erythroid cell diseases Download PDF

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Publication number
WO2004032973A3
WO2004032973A3 PCT/IT2003/000611 IT0300611W WO2004032973A3 WO 2004032973 A3 WO2004032973 A3 WO 2004032973A3 IT 0300611 W IT0300611 W IT 0300611W WO 2004032973 A3 WO2004032973 A3 WO 2004032973A3
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WO
WIPO (PCT)
Prior art keywords
sns
insulator
sea urchin
gene therapy
erythroid cell
Prior art date
Application number
PCT/IT2003/000611
Other languages
French (fr)
Other versions
WO2004032973A2 (en
Inventor
Aurelio Maggio
Santa Anna Acuto
Marzo Rosalba Di
Giovanni Spinelli
Original Assignee
Aurelio Maggio
Santa Anna Acuto
Marzo Rosalba Di
Giovanni Spinelli
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 Aurelio Maggio, Santa Anna Acuto, Marzo Rosalba Di, Giovanni Spinelli filed Critical Aurelio Maggio
Priority to AU2003279536A priority Critical patent/AU2003279536A1/en
Publication of WO2004032973A2 publication Critical patent/WO2004032973A2/en
Publication of WO2004032973A3 publication Critical patent/WO2004032973A3/en

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    • 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
    • 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
    • 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
    • C07K14/43504—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

The invention concerns the application of the sea urchin SNS insulator, or of its fundamental sequences, or of a longer sequence which includes them, to the construction of viral vectors enabling an efficient and lasting ex­pression of the transgene delivered in the target cells, for the treatment of diseases caused by gene defects and, in particular, blood diseases affecting the erythroid line.
PCT/IT2003/000611 2002-10-09 2003-10-09 Use of sns insulator of sea urchin for the gene therapy of erythroid cell diseases WO2004032973A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2003279536A AU2003279536A1 (en) 2002-10-09 2003-10-09 Use of sns insulator of sea urchin for the gene therapy of erythroid cell diseases

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
ITRM2002A000511 2002-10-09
IT000511A ITRM20020511A1 (en) 2002-10-09 2002-10-09 USE OF THE SEA URCHIN INSULATOR SNS FOR GENE THERAPY OF DISEASES OF Erythroid Cells.

Publications (2)

Publication Number Publication Date
WO2004032973A2 WO2004032973A2 (en) 2004-04-22
WO2004032973A3 true WO2004032973A3 (en) 2004-09-16

Family

ID=11456518

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IT2003/000611 WO2004032973A2 (en) 2002-10-09 2003-10-09 Use of sns insulator of sea urchin for the gene therapy of erythroid cell diseases

Country Status (3)

Country Link
AU (1) AU2003279536A1 (en)
IT (1) ITRM20020511A1 (en)
WO (1) WO2004032973A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3287142B1 (en) * 2011-04-08 2021-08-04 University Of Leicester Methods for treating conditions associated with masp-2 dependent complement activation

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1217071A1 (en) * 2000-12-22 2002-06-26 Institut National De La Recherche Agronomique (Inra) Position-independent and tissue specific expression of a transgene in milk of transgenic animals

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1217071A1 (en) * 2000-12-22 2002-06-26 Institut National De La Recherche Agronomique (Inra) Position-independent and tissue specific expression of a transgene in milk of transgenic animals

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
AKASAKA KOJI ET AL: "Upstream element of the sea urchin arylsulfatase gene serves as an insulator", CELLULAR AND MOLECULAR BIOLOGY (NOISY-LE-GRAND), vol. 45, no. 5, July 1999 (1999-07-01), pages 555 - 565, XP009032717 *
DATABASE EBI retrieved from EBI Database accession no. Y09062 *
DI SIMONE PAOLA ET AL: "The sea urchin sns insulator blocks CMV enhancer following integration in human cells", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 284, no. 4, 22 June 2001 (2001-06-22), pages 987 - 992, XP002285881, ISSN: 0006-291X *
PALLA FRANCO ET AL: "Enhancer blocking activity located near the 3' end of the sea urchin early H2A histone gene", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, vol. 94, no. 6, 1997, pages 2272 - 2277, XP002285882, ISSN: 0027-8424 *

Also Published As

Publication number Publication date
ITRM20020511A1 (en) 2004-04-10
AU2003279536A8 (en) 2004-05-04
ITRM20020511A0 (en) 2002-10-09
WO2004032973A2 (en) 2004-04-22
AU2003279536A1 (en) 2004-05-04

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