Publications

Resolution of hepatic fibrosis after zinc-finger nuclease-mediated gene editing in the PiZ mouse model of human alpha-1-antitrypsin deficiency liver disease
March 7th, 2023
First-in-human in vivo genome editing via AAV-zinc finger nucleases for mucopolysaccharidosis I/II and hemophilia B
November 1st, 2022
Regulatory Consideration for the Nonclinical Safety Assessment of Gene Therapies
October 4th, 2022
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation
October 4th, 2022
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy
October 4th, 2022
Human Leukocyte Antigen Class I Molecule A* 02-Chimeric Antigen Receptor Regulatory T Cells in Renal Transplantation
October 4th, 2022
Pluripotent stem cell-based cell therapies: Current applications and future prospects
October 4th, 2022
Overview of Analytics Needed to Support a Robust Gene Therapy Manufacturing Process
January 19th, 2022
Anion-Exchange HPLC Assay for Separation and Quantification of Empty and Full Capsids in Multiple Adeno-Associated Virus Serotypes
April 23rd, 2021
ZFN-Mediated In Vivo Gene Editing in Hepatocytes Leads to Supraphysiologic α-Gal A Activity and Effective Substrate Reduction in Fabry Mice
March 26th, 2021