Adeno-Associated Virus Vectors (AAV) Expressing Phenylalanine Hydroxylase (PAH) - Letter to the Editor

Abstract
Recent articles have appeared in the literature reporting use of adeno-associated virus vectors (AAV) expressing phenylalanine hydroxylase in animal trials and suggesting its use in treatment of phenylketonuria (PKU) as a form of gene therapy However, agents used in gene therapy to deliver genes are not site-specific and DNA is may be put in the wrong place, causing damage to the organism. The adverse immunogenicity of AAVs also needs to be reconsidered. This letter is written to discuss present unreadiness for Phase 1 clinical trials of gene therapy of PKU.