Gene Therapy Review: Gene and Cell Therapy Resource

INHERINET (Gene Therapy of Hematopoietic Stem Cells for Inherited Diseases), an initiative founded by seven European research institutions, accumulates the expertise of biomedical scientists who have a strong background in the development of all platform technologies required for successful gene therapy:

• State-of -the-art design and production of retroviral and lentiviral gene transfer vectors
• Basic and applied biology of hematopoiesis and cell transplantation
• Relevant experimental models and clinical expertise for the treatment of inherited hematopoietic disorders
• Diagnostic tools to evaluate the safety and efficiency of gene therapeutic interventions in preclinical models and clinical applications

INHERINET focuses on the design and preparation for clinical trial through research and preclinical evaluation for:

• Diamond-Blackfan anemia
• Mucopolysaccharidosis VI - Maroteaux-Lamy
• Gaucher's disease

These disease entities have been chosen as representative examples for diseases that with the technology applied by INHERINET will be accessible to gene therapy approaches with curative intent.

Inherinet (QLK3-CT-2001-00427) is a project supported by the Quality of Life programme of the 5^th Framework Programme of the European Commission