Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges

Por Raj, Deepak, Davidoff, Andrew M, Nathwani, Amit C
Publicado em 2011

Our Journey to Successful Gene Therapy for Hemophilia B

Por Nathwani, Amit C., Nienhuis, Arthur W., Davidoff, Andrew M.
Publicado no Hum Gene Ther (2014)

Progress towards Gene Therapy for Haemophilia B

Por Patel, Nishil, Reiss, Ulrike, Davidoff, Andrew M., Nathwani, Amit C.
Publicado em 2014

AAV Vector Biology in Primates: Finding the Missing Link?

Por Herzog, Roland W, Davidoff, Andrew M, Markusic, David M, Nathwani, Amit C
Publicado em 2011

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

Por Cancio, Maria I, Reiss, Ulrike M, Nathwani, Amit C, Davidoff, Andrew M, Gray, John T
Publicado em 2013

Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV mediated gene transfer with a non-depleting CD4 antibody and cyclosporine

Por McIntosh, Jenny, Cochrane, Melanie, Cobbold, Stephen, Waldmann, Herman, Davidoff, Andrew M., Nathwani, Amit C.
Publicado em 2011

Systemic Errors in Quantitative Polymerase Chain Reaction Titration of Self-Complementary Adeno-Associated Viral Vectors and Improved Alternative Methods

Por Fagone, Paolo, Wright, J. Fraser, Nathwani, Amit C., Nienhuis, Arthur W., Davidoff, Andrew M., Gray, John T.
Publicado em 2012

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

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

AAV-mediated local delivery of interferon-β for the treatment of retinoblastoma in preclinical models

Por Shih, Chie-Schin, Laurie, Nikia, Holzmacher, Jeremy, Hu, Yunyu, Nathwani, Amit C., Davidoff, Andrew M., Dyer, Michael A.
Publicado em 2009

Targeting multiple angiogenic pathways for the treatment of neuroblastoma

Por Williams, Regan F., Myers, Adrianne L., Sims, Thomas L., Ng, Catherine Y., Nathwani, Amit C., Davidoff, Andrew M.
Publicado em 2010

ENHANCING TRANSDUCTION OF THE LIVER BY ADENO-ASSOCIATED VIRAL VECTORS

Por Nathwani, Amit C., Cochrane, Melanie, McIntosh, Jenny, Ng, Catherine Y.C., Zhou, Junfang, Gray, John T., Davidoff, Andrew M.
Publicado em 2008

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

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

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

Por Piras, Bryan A, Drury, Jason E, Morton, Christopher L, Spence, Yunyu, Lockey, Timothy D, Nathwani, Amit C, Davidoff, Andrew M, Meagher, Michael M
Publicado no Mol Ther Methods Clin Dev (2016)

Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman prima...

Por Nathwani, Amit C., Gray, John T., McIntosh, Jenny, Ng, Catherine Y. C., Zhou, Junfang, Spence, Yunyu, Cochrane, Melanie, Gray, Elaine, Tuddenham, Edward G. D., Davidoff, Andrew M.
Publicado em 2007

Preferential Targeting of Disseminated Liver Tumors Using a Recombinant Adeno-Associated Viral Vector

Por Della Peruta, Marco, Badar, Adam, Rosales, Cecilia, Chokshi, Shilpa, Kia, Azadeh, Nathwani, Devhrut, Galante, Eva, Yan, Ran, Arstad, Erik, Davidoff, Andrew M., Williams, Roger, Lythgoe, Mark F., Nathwani, Amit C.
Publicado no Hum Gene Ther (2015)

Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial

Por Allay, James A., Sleep, Susan, Long, Scott, Tillman, David M., Clark, Rob, Carney, Gael, Fagone, Paolo, McIntosh, Jenny H., Nienhuis, Arthur W., Davidoff, Andrew M., Nathwani, Amit C., Gray, John T.
Publicado em 2011

Continuous Local Delivery of Interferon-β Stabilizes Tumor Vasculature in an Orthotopic Glioblastoma Xenograft Resection Model

Por Denbo, Jason W., Williams, Regan F., Orr, W. Shannon, Sims, Thomas L., Ng, Catherine Y., Zhou, Junfang, Spence, Yunyu, Morton, Christopher L., Nathwani, Amit C., Duntsch, Christopher, Pfeffer, Lawrence M., Davidoff, Andrew M.
Publicado em 2011

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman...

Por Nathwani, Amit C., Gray, John T., Ng, Catherine Y. C., Zhou, Junfang, Spence, Yunyu, Waddington, Simon N., Tuddenham, Edward G. D., Kemball-Cook, Geoffrey, McIntosh, Jenny, Boon-Spijker, Mariette, Mertens, Koen, Davidoff, Andrew M.
Publicado em 2006

Interferon-β Sensitizes Neuroblastoma to the Anti-Tumor Activity of Temozolomide by Modulating O(6)-Methylguanine-DNA Methyltransferase Expression

Por Rosati, Shannon F., Williams, Regan F., Nunnally, Lindsey C., McGee, Mackenzie C., Sims, Thomas L., Tracey, Lorraine, Zhou, Junfang, Fan, Meiyun, Ng, Catherine Y., Nathwani, Amit C., Stewart, Clinton F., Pfeffer, Lawrence M., Davidoff, Andrew M.
Publicado em 2008

Improved intratumoral oxygenation through vascular normalization increases glioma sensitivity to ionizing radiation

Por McGee, Mackenzie C., Hamner, J. Blair, Williams, Regan F., Rosati, Shannon F., Sims, Thomas L., Ng, Catherine Y., Gaber, M. Waleed, Calabrese, Christopher, Wu, Jianrong, Nathwani, Amit C., Duntsch, Christopher, Merchant, Thomas E., Davidoff, Andrew M.
Publicado em 2010