Publication
5/05/2021
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
Publication
2/12/2020
A Mutation-Independent CRISPR-Cas9-Mediated Gene Targeting Approach to Treat a Murine Model of Ornithine Transcarbamylase Deficiency
Wang L, Yang Y, Breton C, Bell P, Li M, Zhang J, Che Y, Saveliev A, He Z, White J, Latshaw C, Xu C, McMenamin D, Yu H, Morizono H, Batshaw ML, Wilson JM
Science Advances
Publication
6/01/2018
Preparation of Nonhuman Primate Eyes for Histological Evaluation After Retinal Gene Transfer.
Bell P, Yu H, Kuntz L, Ahonkhai O, Tretiakova A, Limberis MP, Wilson JM
Human Gene Therapy Methods
Publication
1/01/2018
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
Publication
4/01/2017
AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.
Wang L, Bell P, Morizono H, He Z, Pumbo E, Yu H, White J, Batshaw ML, Wilson JM
Molecular Genetics And Metabolism
Publication
12/01/2016
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