Europe PMC

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Abstract 


This unit is devoted to safety issues that must be considered when generating and working with vectors for gene therapy. A short description of the relevant regulatory bodies and mechanisms is followed by a general discussion of containment and safety procedures. This is followed by vector-specific guidelines for nine commonly used vector systems, including adenovirus, vaccinia, HSV-1, lentivirus, and nonviral vectors. Recent tragic experiences with retroviral integration near oncogene loci demonstrate the surprise effects of successful gene therapy. The unit also outlines the problems encountered when taking a promising vector to clinical trials.

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