Developments in the treatment of hemophilia B: focus on emerging gene therapy

by Cancio, Maria I, Reiss, Ulrike M, Nathwani, Amit C, Davidoff, Andrew M, Gray, John T
Published 2013

Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy

by Sack, Brandon K., Merchant, Sherin, Markusic, David M., Nathwani, Amit C., Davidoff, Andrew M., Byrne, Barry J., Herzog, Roland W.
Published 2012

A Systematic Evaluation of Integration Free Reprogramming Methods for Deriving Clinically Relevant Patient Specific Induced Pluripotent Stem (iPS) Cells

by Goh, Pollyanna A., Caxaria, Sara, Casper, Catharina, Rosales, Cecilia, Warner, Thomas T., Coffey, Pete J., Nathwani, Amit C.
Published 2013

Distribution of AAV8 particles in cell lysates and culture media changes with time and is dependent on the recombinant vector

by Piras, Bryan A, Drury, Jason E, Morton, Christopher L, Spence, Yunyu, Lockey, Timothy D, Nathwani, Amit C, Davidoff, Andrew M, Meagher, Michael M
Published 2016

AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage

by Binny, Christopher, McIntosh, Jenny, Della Peruta, Marco, Kymalainen, Hanna, Tuddenham, Edward G. D., Buckley, Suzanne M. K., Waddington, Simon N., McVey, John H., Spence, Yunyu, Morton, Christopher L., Thrasher, Adrian J., Gray, John T., Castellino, Francis J., Tarantal, Alice F., Davidoff, Andrew M., Nathwani, Amit C.
Published 2012