Development of a GMP-compliant packaging cell line for making lentiviral vectors

Development of a GMP-compliant packaging cell line for making lentiviral vectors

In classical gene therapy, genetic information is introduced into cells to alleviate cellular malfunctions, for example. This introduction of genetic information is generally achieved with the help of certain viruses ("MMLV-based retroviruses") which have some disadvantages, however. During this project, a cell line suitable for manufacturing ("packaging cell line") is to be developed and manufactured that can produce not MMLV-based retroviruses, but a different type of virus, so-called ‘lentiviruses’. A fundamental difference between the two types of virus is the source of the original virus-isolates: the MMLV-based virus comes from a mouse, whereas the HIV-based retrovirus comes from a human. In the viruses used for gene therapy, the virus-specific genes have been replaced by the respective therapeutic gene. The virus used in therapy contains practically only the sequences required for viral replication, such as a packaging signal. All other viral genetic information has been removed, which is why this technology has been used successfully in hospitals for years. In gene therapy, lentiviruses may be superior to MMLV-based retroviruses because they can also introduce  their genetic information into quiescent, non-dividing cells. If successful, correcting a malfunction, e.g. a genetic defect, using gene therapy could lead to permanent elimination of the disease (healing).

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