Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy
Hurlbut, Gregory D, Ziegler, Robin J, Nietupski, Jennifer B, Foley, Joseph W, Woodworth, Lisa A, Meyers, Elizabeth, Bercury, Scott D, Pande, Nilesh N, Souza, David W, Bree, Mark P, Lukason, Michael J, Marshall, John, Cheng, Seng H, Scheule, Ronald K
Published in Molecular therapy (01.11.2010)
Published in Molecular therapy (01.11.2010)
Get full text
Journal Article
Soluble Transforming Growth Factor-beta Type III Receptor Gene Transfection Inhibits Fibrous Airway Obliteration in a Rat Model of Bronchiolitis Obliterans
LIU, MINGYAO, SUGA, MICHIHARU, MACLEAN, ALEXANDRA A, ST. GEORGE, JUDITH A, SOUZA, DAVID W, KESHAVJEE, SHAF
Published in American journal of respiratory and critical care medicine (01.02.2002)
Published in American journal of respiratory and critical care medicine (01.02.2002)
Get full text
Journal Article
AAV2 Vector Harboring a Liver-Restricted Promoter Facilitates Sustained Expression of Therapeutic Levels of α-Galactosidase A and the Induction of Immune Tolerance in Fabry Mice
Ziegler, Robin J, Lonning, Scott M, Armentano, Donna, Li, Chester, Souza, David W, Cherry, Maribeth, Ford, Christine, Barbon, Christine M, Desnick, Robert J, Gao, Guangping, Wilson, James M, Peluso, Richard, Godwin, Simon, Carter, Barrie J, Gregory, Richard J, Wadsworth, Samuel C, Cheng, Seng H
Published in Molecular therapy (01.02.2004)
Published in Molecular therapy (01.02.2004)
Get full text
Journal Article
Demonstration that CFTR is a Chloride Channel by Alteration of its Anion Selectivity
Anderson, Matthew P., Gregory, Richard J., Thompson, Simon, Souza, David W., Paul, Sucharita, Mulligan, Richard C., Smith, Alan E., Welsh, Michael J.
Published in Science (American Association for the Advancement of Science) (12.07.1991)
Published in Science (American Association for the Advancement of Science) (12.07.1991)
Get full text
Journal Article
Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis
Cheng, Seng H., Gregory, Richard J., Marshall, John, Paul, Sucharita, Souza, David W., White, Gary A., O'Riordan, Catherine R., Smith, Alan E.
Published in Cell (16.11.1990)
Published in Cell (16.11.1990)
Get full text
Journal Article
Constitutive Expression of Glucagon-Like Peptide 1 for Gene Therapy of Type 2 Diabetes
Parsons, Geoffrey B, Yu, Dan, Souza, David W, Wu, Hui
Published in Diabetes (New York, N.Y.) (01.06.2005)
Get full text
Published in Diabetes (New York, N.Y.) (01.06.2005)
Journal Article
Maturation and function of cystic fibrosis transmembrane conductance regulator variants bearing mutations in putative nucleotide-binding domains 1 and 2
Gregory, R J, Rich, D P, Cheng, S H, Souza, D W, Paul, S, Manavalan, P, Anderson, M P, Welsh, M J, Smith, A E
Published in Molecular and Cellular Biology (01.08.1991)
Published in Molecular and Cellular Biology (01.08.1991)
Get full text
Journal Article
AAV2 Vector Harboring a Liver-Restricted Promoter Facilitates Sustained Expression of Therapeutic Levels of [alpha]-Galactosidase A and the Induction of Immune Tolerance in Fabry Mice
Ziegler, Robin J, Lonning, Scott M, Armentano, Donna, Li, Chester, Souza, David W, Cherry, Maribeth, d, Christine, Barbon, Christine M, Desnick, Robert J, Gao, Guangping, Wilson, James M, Peluso, Richard, Godwin, Simon, Carter, Barrie J, Gregory, Richard J, Wadsworth, Samuel C, Cheng, Seng H
Published in Molecular therapy (01.02.2004)
Published in Molecular therapy (01.02.2004)
Get full text
Journal Article