AAV vectors for gene correction.
Permanent correction of genetic mutations is the ultimate goal in the treatment of inherited disorders. Although gene therapy protocols have proven successful in several clinical trials that focused on monogenetic diseases, alternative approaches have become the subject of growing interest since two cases of insertional mutagenesis were reported in one of these trials. Gene correction is an alternative technology that aims to repair a mutated gene rather than complement the mutation by adding a wild-type copy of the affected gene. This review focuses on viral approaches to gene correction, especially the use of vectors based on adeno-associated viruses. The pitfalls and prospects, as well as the steps needed to improve and advance the technology for clinical applications, are discussed.
Cathomen
Toni
T
Charité Medical School, Institute of Infectious Diseases, Department of Virology, Hindenburgdamm 27, 12203 Berlin, Germany. toni.cathomen@charite.de
D000818:Animals; D000229:Dependovirus / Q000235:genetics* / Q000378:metabolism; D018390:Gene Targeting; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics / Q000378:metabolism; D006801:Humans