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Stem Cell Related Patent Number US6361997

Title:Genetically modified CD34-negative adherently growing stem cells and their use in gene therapy
Inventors:Huss, Ralf; 83666 Waakirchen, Germany
Summary:This invention introduces genetically modified, adherently growing CD34-negative stem cells. Described herein are very early, genetically modified hematopoietic and mesenchymal stem cells which are negative for the expression of the surface molecule CD34. Methods are provided by which the autologous CD34-negative adherently growing stem cell cultures from the peripheral blood of a patient are applied and efficiently tranfected or infected with genetic constructs, such that the gene products of these genes have been shown to provide long-term substitution for defective or absent proteins or factors in a patient. Therapeutic applications of such autologous stem cells include organ replacement and the transfer of gene therapy constructs for the treatment of immunodeficiency and metabolic disorders.
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).
US Patent Website:Click Here for Full Text of Patent
Title Number:US6361997
Application Number:US2001000743904
Date Filed:24/02/2001
Date Published:26/03/2002
Assignee:None


 
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