David W. Souza

Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis

Seng H. Cheng, Richard J. Gregory, John Marshall et al. · Cell · 1990 · 1.8K citations

Fibrosis, Molecular Physiology, Defective Intracellular Transport +8

Demonstration That CFTR Is a Chloride Channel by Alteration of Its Anion Selectivity

Matthew P. Anderson, Richard J. Gregory, Simon Thompson et al. · Science · 1991 · 1.1K citations

Maturation and function of cystic fibrosis transmembrane conductance regulator variants bearing mutations in putative nucleotide-binding domains 1 and 2.

Richard J. Gregory, Devra P. Rich, Shuk Han Cheng et al. · Molecular and Cellular Biology · 1991 · 247 citations · Full text

Protein Function, Signal Transduction, Molecular Physiology +15

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

Robin J. Ziegler, Scott Lonning, Donna Armentano et al. · Molecular Therapy · 2003 · 135 citations

Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy

Gregory D Hurlbut, Robin J. Ziegler, Jennifer B. Nietupski et al. · Molecular Therapy · 2010 · 134 citations · Full text

Immunology, Pathology, Immunotherapy +16