GroseljU, TansekMZ, BattelinoT. Fifty years of phenylketonuria newborn screening – A great success for many, but what about the rest?. Mol Gen Metab, 2014; 113:8–10.
2.
HardingCO, GillinghamMB, HammanK, ClarkH, Goebel-DaghighiE, BirdA, et al.Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria. Gene Ther, 2006; 13:457–462.
3.
MarkmannS, DeBP, ReidJ, JoseC, RosenbergJB, LeopoldP, et al.Biology of the adrenal gland cortex obviates effective use of adeno-associated virus vectors to treat hereditary adrenal disorders. Hum Gene Ther, 2018Jan9 [Epub ahead of print]; DOI: 10.1089/hum.2017.203.
4.
FlotteTR, AfioneSA, ZeitlinPL. Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am J Respir Cell Mol Biol, 1994; 11:517–521.
5.
AfioneSA, ConradCK, KearnsWG, ChunduruS, AdamsR, ReynoldsTC, et al.In vivo model of adeno-associated virus vector persistence and rescue. J Virol, 1996; 70:3235–3241.
6.
NakaiH, YantSR, StormTA, FuessS, MeuseL, KayMA. Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J Virol, 2001; 75:6969–6976.
