Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia.
Wang L, Muthuramu I, Somanathan S, Zhang H, Bell P, He Z, Yu H, Zhu Y, Tretiakova AP, Wilson JM
Molecular Therapy. Methods & Clinical Development
Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9.
Wang L, Breton C, Warzecha CC, Bell P, Yan H, He Z, White J, Zhu Y, Li M, Buza EL, Jantz D, Wilson JM
Molecular Therapy : The Journal Of The American Society Of Gene Therapy
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates.
Hordeaux J, Buza EL, Jeffrey B, Song C, Jahan T, Yuan Y, Zhu Y, Bell P, Li M, Chichester JA, Calcedo R, Wilson JM
Science Translational Medicine
A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease.
Greig JA, Nordin JML, Smith MK, Ashley SN, Draper C, Zhu Y, Bell P, Buza EL, Wilson JM
Human Gene Therapy. Clinical Development
Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals.
Hinderer C, Bell P, Katz N, Vite CH, Louboutin JP, Bote E, Yu H, Zhu Y, Casal ML, Bagel J, O'Donnell P, Wang P, Haskins ME, Goode T, Wilson JM
Human Gene Therapy
Effects of Self-Complementarity, Codon Optimization, Transgene, and Dose on Liver Transduction with AAV8.
Bell P, Wang L, Chen SJ, Yu H, Zhu Y, Nayal M, He Z, White J, Lebel-Hagan D, Wilson JM
Human Gene Therapy Methods