Gene therapy is an approach to treat different inherited diseases but also conditions like cancer, HIV, Parkinson’s or Alzheimer’s Disease by targeted expression of specific proteins using either viral DNA or RNA vectors, applying missing or defective sequences. For the clinical application of therapeutically used genes it is important to control the biodistribution of the gene vector and its expression. Beside the tissues/organs of interest where the specific expression of a protein is desired also gene expression in other tissues has to be monitored because there the expression of the gene could induce severe side effects. Specific attention has to be paid to possible germ line transmission of the vector.
It is evident that before such therapeutic genes can be used in clinical studies intensive pre-clinical investigations are required. JSW-Research, a GLP-certified Contract Research Organization located in Graz, Austria, is specialized in such safety investigations. JSW developed and validated highly sensitive quantitative polymerase chain reaction procedures (qPCR) to enable the detection of extremely small amounts of the gene of interest in different tissues and body fluids. This involves specialized and organ specific extraction procedures and sophisticated sample processing. These methods have already been used for preclinical studies investigating the therapeutic vector in animal tissues to collect information about biodistribution, time course and level of expression. But JSW also offers a use of these technologies for safety monitoring in first therapeutic human trials, monitoring biodistribution and expression of the therapeutic vector in different body fluids (serum, saliva, urine, semen/seminal fluid).
These investigations help to improve the safety of gene therapy which is an enormous hope for efficacious treatment of different diseases in the near future.
It is evident that before such therapeutic genes can be used in clinical studies intensive pre-clinical investigations are required. JSW-Research, a GLP-certified Contract Research Organization located in Graz, Austria, is specialized in such safety investigations. JSW developed and validated highly sensitive quantitative polymerase chain reaction procedures (qPCR) to enable the detection of extremely small amounts of the gene of interest in different tissues and body fluids. This involves specialized and organ specific extraction procedures and sophisticated sample processing. These methods have already been used for preclinical studies investigating the therapeutic vector in animal tissues to collect information about biodistribution, time course and level of expression. But JSW also offers a use of these technologies for safety monitoring in first therapeutic human trials, monitoring biodistribution and expression of the therapeutic vector in different body fluids (serum, saliva, urine, semen/seminal fluid).
These investigations help to improve the safety of gene therapy which is an enormous hope for efficacious treatment of different diseases in the near future.
