Gene therapy for hemophilia

por Nathwani, Amit C.
Publicado en Hematology Am Soc Hematol Educ Program (2019)

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 2011

Our Journey to Successful Gene Therapy for Hemophilia B

por Nathwani, Amit C., Nienhuis, Arthur W., Davidoff, Andrew M.
Publicado en Hum Gene Ther (2014)

Gene Therapy for Hemophilia

por Nienhuis, Arthur W., Nathwani, Amit C., Davidoff, Andrew M.
Publicado en Mol Ther (2017)

Mbd3 Promotes Reprogramming of Primary Human Fibroblasts

por Jaffer, Sajjida, Goh, Pollyanna, Abbasian, Mahnaz, Nathwani, Amit C
Publicado en Int J Stem Cells (2018)

Progress towards Gene Therapy for Haemophilia B

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

AAV Vector Biology in Primates: Finding the Missing Link?

por Herzog, Roland W, Davidoff, Andrew M, Markusic, David M, Nathwani, Amit C
Publicado 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 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 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 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 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 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 2010

Modification of a Constitutive to Glucose-Responsive Liver-Specific Promoter Resulted in Increased Efficacy of Adeno-Associated Virus Serotype 8-Insulin Gene Therapy of Diabetic Mi...

por Sia, Kian Chuan, Fu, Zhen Ying, Calne, Roy Y., Nathwani, Amit C., Lee, Kok Onn, Gan, Shu Uin
Publicado en Cells (2020)

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 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 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 en 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 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 en 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 2011