Analyzing the Genotoxicity of Retroviral Vectors in Hematopoietic Cell Gene Therapy

Analyzing the Genotoxicity of Retroviral Vectors in Hematopoietic Cell Gene Therapy

Blog Article

Retroviral vectors, including those derived from gammaretroviruses and lentiviruses, have found their way into the clinical arena and demonstrated remarkable efficacy for the treatment of immunodeficiencies, leukodystrophies, and globinopathies.Despite these successes, gene therapy unfortunately also has had to face severe adverse events in the form of leukemias and myelodysplastic syndromes, related to the semi-random SORE THROAT SPRAY vector integration into the host cell genome that caused deregulation of neighboring proto-oncogenes.Although improvements in vector design clearly lowered the risk of this insertional mutagenesis, analysis of potential genotoxicity and the consequences of vector integration remain important parameters for basic and translational research and most importantly for the clinic.

Here, we review current assays to analyze NEFF N50 HLAWD53N0B Built-In Solo Microwave 900 Watt 25 L - Black biodistribution and genotoxicity in the pre-clinical setting and describe tools to monitor vector integration sites in vector-treated patients as a biosafety readout.