Retrovirus mediated hematopoietic gene therapy: A European regulatory perspective with special focus on the situation in Germany
Year: 2010
Type: article
Abstract: a preparatory "conditioning" by cytoreductive treatment may be required to promote engraftment.Stable gene transfer, and therefore long-term genetic modification, is achieved by gene vectors, which integrate their genetic material and the therapeutic gene respectively into the chromatin of the target cells.Unlike classical pharmaceutical drug treatment, gene therapy is an approach highly specific to the patient and the disease.Parameters like cell source and origin, vector type, therapeutic dose, route of administration and, in particular, the trans- SummaryRetrovirus mediated gene therapy has already proven to be more than just a theoretical option to treat patients with severe genetic defects.Clinical gene therapy trials of X-linked severe combined immunodeficiency or adenosine deaminase deficiency have demonstrated the success and potential benefit of the therapy.Nevertheless, the complexity of the therapeutic products and their biological origin, as well as virus-related safety concerns require the need of a strict regulatory framework in order to guarantee the quality of the individual products and safety of the patients.The aim of this review is to give an overview of the rapidly evolving regulatory framework of Advanced Therapy Medicinal Products in Europe.We will summarize the most important regulatory documents to be considered before entering the clinical development phase-not only from a German but also from a European perspective.
Source: CyberLeninK (CyberLeninka)
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: green
OpenAlex ID: W2548818469