Manufacturing and characterizing AAV-based vectors for use in clinical studies.
Recombinant adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great promise for human gene therapy. A major challenge for translation of promising research to clinical development is the manufacture and certification of AAV vectors for clinical use. This review summarizes relevant aspects of current Good Manufacturing Practice, focusing on considerations and challenges specific for recombinant AAV.
Wright
J F
JF
Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA. wrightf@email.chop.edu
D000229:Dependovirus / Q000235:genetics*; D005818:Genetic Engineering / Q000592:standards; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics*; D006801:Humans; D007221:Industry; D011786:Quality Control; D011994:Recombinant Proteins / Q000235:genetics
D011994:Recombinant Proteins