Genetically modified CD34-negative adherently growing stem cells and their use in gene therapy

• US 6,361,997 B1
• Filed: 02/24/2001
• Issued: 03/26/2002
• Est. Priority Date: 07/24/1998
• Status: Expired due to Term

• Current Assignee: Apceth GmbH & Co. KG (Resonac Holdings Corp.)
• Original Assignee: Ralf Huss
• Inventors: Huss, Ralf
• Primary Examiner(s): KETTER, JAMES S
• Application Number:
  US09/743,904
• Time in Patent Office: 395 Days
• Field of Search: 514/44, 435/372, 435/372.1, 435/372.2, 435/372.3, 435/6
• US Class Current: 435/372
• CPC Class Codes: A61K 2035/124 the cells being hematopoietic, bone marrow derived or blood cells
• CPC Class Codes: A61K 48/00 Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
• CPC Class Codes: A61P 31/04 Antibacterial agents
• CPC Class Codes: A61P 43/00 Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
• CPC Class Codes: A61P 5/50 for increasing or potentiating the activity of insulin
• CPC Class Codes: A61P 7/04 Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
• CPC Class Codes: C12N 2501/23 Interleukins [IL]
• CPC Class Codes: C12N 2510/00 Genetically modified cells
• CPC Class Codes: C12N 5/0647 Haematopoietic stem cells; Uncommitted or multipotent progenitors

Abstract

The invention relates to the use of genetically modified, very early haematopoietic and mesenchymal stem cells (negative for the expression of the surface molecule CD34) in the individual gene therapy of mono- or oligogenetic diseases or in cell therapy. Autologous CD34-negative adherently growing stem cell cultures from the peripheral blood of the patient are applied and efficiently tranfected or infected with genetic constructs. The gene products of these genes should substitute defective or absent proteins or factors in the patient organism in the long term. After expansion, the autologous stem cells can also be used for cell therapy (organ replacement therapy).

Claims

1. Genetically modified CD34-negative adherently growing stem cells.

2. Genetically modified CD34-negative adherently growing stem cells according to claim 1, further comprising a construct selected from the group consisting of a retroviral construct an EBV construct and an adenovirus-associated construct.