Gene therapy for hemophilia

ανά Nathwani, Amit C.
Τόπος έκδοσης Hematology Am Soc Hematol Educ Program (2019)

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

ανά Raj, Deepak, Davidoff, Andrew M, Nathwani, Amit C
Έκδοση 2011

Our Journey to Successful Gene Therapy for Hemophilia B

ανά Nathwani, Amit C., Nienhuis, Arthur W., Davidoff, Andrew M.
Τόπος έκδοσης Hum Gene Ther (2014)

Gene Therapy for Hemophilia

ανά Nienhuis, Arthur W., Nathwani, Amit C., Davidoff, Andrew M.
Τόπος έκδοσης Mol Ther (2017)

Mbd3 Promotes Reprogramming of Primary Human Fibroblasts

ανά Jaffer, Sajjida, Goh, Pollyanna, Abbasian, Mahnaz, Nathwani, Amit C
Τόπος έκδοσης Int J Stem Cells (2018)

Progress towards Gene Therapy for Haemophilia B

ανά Patel, Nishil, Reiss, Ulrike, Davidoff, Andrew M., Nathwani, Amit C.
Έκδοση 2014

AAV Vector Biology in Primates: Finding the Missing Link?

ανά Herzog, Roland W, Davidoff, Andrew M, Markusic, David M, Nathwani, Amit C
Έκδοση 2011

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

ανά Cancio, Maria I, Reiss, Ulrike M, Nathwani, Amit C, Davidoff, Andrew M, Gray, John T
Έκδοση 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

ανά McIntosh, Jenny, Cochrane, Melanie, Cobbold, Stephen, Waldmann, Herman, Davidoff, Andrew M., Nathwani, Amit C.
Έκδοση 2011

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

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

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

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

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

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

Targeting multiple angiogenic pathways for the treatment of neuroblastoma

ανά Williams, Regan F., Myers, Adrianne L., Sims, Thomas L., Ng, Catherine Y., Nathwani, Amit C., Davidoff, Andrew M.
Έκδοση 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...

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

ENHANCING TRANSDUCTION OF THE LIVER BY ADENO-ASSOCIATED VIRAL VECTORS

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

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

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

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

ανά Piras, Bryan A, Drury, Jason E, Morton, Christopher L, Spence, Yunyu, Lockey, Timothy D, Nathwani, Amit C, Davidoff, Andrew M, Meagher, Michael M
Τόπος έκδοσης 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...

ανά 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.
Έκδοση 2007

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

ανά 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.
Τόπος έκδοσης Hum Gene Ther (2015)

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

ανά 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.
Έκδοση 2011