WO1999062562A1 - Implant prothetique et techniques d'utilisation aux fins de l'expression de genes therapeutiques - Google Patents

Implant prothetique et techniques d'utilisation aux fins de l'expression de genes therapeutiques Download PDF

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Publication number
WO1999062562A1
WO1999062562A1 PCT/US1999/012116 US9912116W WO9962562A1 WO 1999062562 A1 WO1999062562 A1 WO 1999062562A1 US 9912116 W US9912116 W US 9912116W WO 9962562 A1 WO9962562 A1 WO 9962562A1
Authority
WO
WIPO (PCT)
Prior art keywords
cells
gene product
prosthetic
chamber
cell
Prior art date
Application number
PCT/US1999/012116
Other languages
English (en)
Inventor
William R. A. Osborne
Daniel J. Lejnieks
Original Assignee
University Of Washington
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 University Of Washington filed Critical University Of Washington
Priority to CA002330172A priority Critical patent/CA2330172A1/fr
Priority to AU44107/99A priority patent/AU4410799A/en
Priority to EP99927128A priority patent/EP1083935A1/fr
Priority to DE1083935T priority patent/DE1083935T1/de
Publication of WO1999062562A1 publication Critical patent/WO1999062562A1/fr
Priority to GR20010300045T priority patent/GR20010300045T1/el

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/1816Erythropoietin [EPO]
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system

Definitions

  • the methods of the invention implant under the tunica serosa genetically modified vascular smooth muscle cells for stable long-term cell engraftment expression of Epo.
  • Smooth muscle cells are present at this location and advantageously potentiate the engraftment efficiency and long-term survival of the transplanted cells because they are a normal constituent of the implanted area.
  • autologous transduced cells are injected into a chamber created from a polytetrafluoroethylene (PTFE) ring placed between the serosa and muscularis of the stomach.
  • PTFE polytetrafluoroethylene
  • a measurable effect on a biological or biochemical component of the pathway due to the secreted gene product is sufficient to render the number of implanted cells within the definition of an effective amount as used herein.
  • cell populations between about 10 ' and 10 " are sufficient number of cells that can be implanted and become engrafted so as to secrete a gene product at a level which will reduce one or more symptoms of the target disease.
  • a cell population greater than about 10', preferably greater than about 10% and more preferably greater than about 10 or more cells when implanted in an individual can provide therapeutic effects in vivo .
  • the invention provides a method of systemic delivery of a gene product where cells secreting the gene product are smooth muscle cells or fibroblasts.
  • the gene product can be constitutively secreted, or alternatively, it can be secteted by mducible regulation .
  • Interleukms are a class of immune-related cytokines which can be used to treat a wide variety of diseases and pathological conditions. These molecules function, for example, in immune cell regulation and can be used to augment immune responses for the treatment of immune disorders, including autoimmune diseases, as well as for the treatment of cancer.
  • regulatory T cells can be stimulated with interleukms to augment their ability to induce or inhibit an immune response.
  • immune cells can be stimulated with interleukms to facilitate immune responses against the pathological cells or to increase the effectiveness of other therapies.

Abstract

Cette invention a trait à une technique d'administration systémique d'un produit génique à un individu. Cette technique consiste à implanter dans la paroi du tractus digestif un enceinte prothétique renfermant une quantité efficace de cellules sécrétant le produit génique. Il est possible d'implanter cette enceinte dans une couche de tissu conjonctif ou à proximité de celle-ci, par exemple entre la tunique séreuse et la tunique musculaire de la paroi du tractus digestif. Le produit génique à administrer peut être choisi dans le groupe constitué par des cytokines, des hormones, des facteurs de croissance et des facteurs de coagulation sanguine au nombre desquels, par exemple, l'érythropoïétine ou l'insuline. L'expression du produit génique peut être constitutive ou inductible. Cette invention fournit, de la sorte, une méthode de traitement d'une pathologie liée à la carence d'un produit génique. Cette méthode consiste à implanter une enceinte prothétique renfermant une quantité efficace de cellules exprimant des niveaux thérapeutiques du produit génique manquant.
PCT/US1999/012116 1998-06-02 1999-06-01 Implant prothetique et techniques d'utilisation aux fins de l'expression de genes therapeutiques WO1999062562A1 (fr)

Priority Applications (5)

Application Number Priority Date Filing Date Title
CA002330172A CA2330172A1 (fr) 1998-06-02 1999-06-01 Implant prothetique et techniques d'utilisation aux fins de l'expression de genes therapeutiques
AU44107/99A AU4410799A (en) 1998-06-02 1999-06-01 Prosthetic implant and methods of use for therapeutic gene expression
EP99927128A EP1083935A1 (fr) 1998-06-02 1999-06-01 Implant prothetique et techniques d'utilisation aux fins de l'expression de genes therapeutiques
DE1083935T DE1083935T1 (de) 1998-06-02 1999-06-01 Prosthetisches implantat und verfahren zur verwendung für therapeutische genexpression
GR20010300045T GR20010300045T1 (en) 1998-06-02 2001-08-31 Prosthetic implant and methods of use for therapeutic gene expression

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US8765998P 1998-06-02 1998-06-02
US60/087,659 1998-06-02

Publications (1)

Publication Number Publication Date
WO1999062562A1 true WO1999062562A1 (fr) 1999-12-09

Family

ID=22206492

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1999/012116 WO1999062562A1 (fr) 1998-06-02 1999-06-01 Implant prothetique et techniques d'utilisation aux fins de l'expression de genes therapeutiques

Country Status (7)

Country Link
EP (1) EP1083935A1 (fr)
AU (1) AU4410799A (fr)
CA (1) CA2330172A1 (fr)
DE (1) DE1083935T1 (fr)
ES (1) ES2157874T1 (fr)
GR (1) GR20010300045T1 (fr)
WO (1) WO1999062562A1 (fr)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000006204A2 (fr) * 1998-07-28 2000-02-10 Advanced Tissue Sciences, Inc. Cellules genetiquement modifiees et tissus

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995014785A1 (fr) * 1993-11-23 1995-06-01 Rhone-Poulenc Rorer S.A. Composition pour la production de produits therapeutiques in vivo
WO1995025547A1 (fr) * 1994-03-24 1995-09-28 University Of Washington Dispositif et methodes d'implantation de cellules ayant subi une transduction
JPH0833472A (ja) * 1994-07-25 1996-02-06 Terumo Corp 細胞リザーバー
WO1996040175A1 (fr) * 1995-06-07 1996-12-19 Advanced Tissue Sciences, Inc. Systeme de culture tridimensionnel base sur les cellules du stroma et destine a la formation de tubes, de tendons, de ligaments et de structures correctives

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995014785A1 (fr) * 1993-11-23 1995-06-01 Rhone-Poulenc Rorer S.A. Composition pour la production de produits therapeutiques in vivo
WO1995025547A1 (fr) * 1994-03-24 1995-09-28 University Of Washington Dispositif et methodes d'implantation de cellules ayant subi une transduction
JPH0833472A (ja) * 1994-07-25 1996-02-06 Terumo Corp 細胞リザーバー
WO1996040175A1 (fr) * 1995-06-07 1996-12-19 Advanced Tissue Sciences, Inc. Systeme de culture tridimensionnel base sur les cellules du stroma et destine a la formation de tubes, de tendons, de ligaments et de structures correctives

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
DATABASE WPI Section Ch Week 199615, Derwent World Patents Index; Class A96, AN 1996-145932, XP002121707 *
NAFFAKH N ET AL: "SUSTAINED DELIVERY OF ERYTHROPOIETIN IN MICE BY GENETICALLY MODIFIED SKIN FIBROBLASTS", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, vol. 92, 1 April 1995 (1995-04-01), pages 3194 - 3198, XP002032008, ISSN: 0027-8424 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000006204A2 (fr) * 1998-07-28 2000-02-10 Advanced Tissue Sciences, Inc. Cellules genetiquement modifiees et tissus
WO2000006204A3 (fr) * 1998-07-28 2000-05-04 Advanced Tissue Sciences Inc Cellules genetiquement modifiees et tissus

Also Published As

Publication number Publication date
DE1083935T1 (de) 2001-12-20
EP1083935A1 (fr) 2001-03-21
AU4410799A (en) 1999-12-20
CA2330172A1 (fr) 1999-12-09
GR20010300045T1 (en) 2001-08-31
ES2157874T1 (es) 2001-09-01

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