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Human Gene Therapy Methods|December 2, 2016
Effects of Self-Complementarity, Codon Optimization, Transgene, and Dose on Liver Transduction with AAV8Peter Bell, Lili Wang, Shu-Jen Chen, et al.Blood|April 13, 2019
CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX-knockout miceLili Wang, Yang Yang, Camilo Ayala Breton, et al.Nature Biotechnology|February 2, 2016
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn miceYang Yang, Lili Wang, Peter Bell, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|February 20, 2021
Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9Lili Wang, Camilo Breton, Claude C Warzecha, et al.Nature Biotechnology|November 6, 2023
Integrated vector genomes may contribute to long-term expression in primate liver after AAV administrationJenny A Greig, Kelly M Martins, Camilo Breton, et al.Science Advances|February 26, 2020
A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiencyLili Wang, Yang Yang, Camilo Breton, et al.Nature Biotechnology|July 10, 2018
Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterolLili Wang, Jeff Smith, Camilo Breton, et al.Molecular Therapy. Methods & Clinical Development|February 25, 2022
Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expressionLili Wang, Claude C Warzecha, Alexander Kistner, et al.Pageof 2