Hairpin end conformation of adeno-associated virus (AAV) genome determines interactions with DNA repair pathways

por Cataldi, Marcela P., McCarty, Douglas M.
Publicado 2012

Peripheral Nervous System Neuropathology and Progressive Sensory Impairments in a Mouse Model of Mucopolysaccharidosis IIIB

por Fu, Haiyan, Bartz, Julianne D., Stephens, Robert L., McCarty, Douglas M.
Publicado 2012

Durability of transgene expression after rAAV gene therapy

por Muhuri, Manish, Levy, Daniel I., Schulz, Martin, McCarty, Douglas, Gao, Guangping
Publicado 2022

Mucopolysaccharidosis IIIB, a lysosomal storage disease, triggers a pathogenic CNS autoimmune response

por Killedar, Smruti, DiRosario, Julianne, Divers, Erin, Popovich, Phillip G, McCarty, Douglas M, Fu, Haiyan
Publicado 2010

Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting

por Velazquez, Victoria M., Meadows, Aaron S., Pineda, Ricardo J., Camboni, Marybeth, McCarty, Douglas M., Fu, Haiyan
Publicado 2017

Immunogenicity and inflammatory properties of respiratory syncytial virus attachment G protein in cotton rats

por Martinez, Margaret E., Capella Gonzalez, Cristina, Huey, Devra, Peeples, Mark E., McCarty, Douglas, Niewiesk, Stefan
Publicado 2021

Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice

por Fu, Haiyan, Zaraspe, Kim, Murakami, Naoko, Meadows, Aaron S., Pineda, Ricardo J., McCarty, Douglas M., Muenzer, Joseph
Publicado 2018

Amyloidosis, Synucleinopathy, and Prion Encephalopathy in a Neuropathic Lysosomal Storage Disease: The CNS-Biomarker Potential of Peripheral Blood

por Naughton, Bartholomew J., Duncan, F. Jason, Murrey, Darren, Ware, Tierra, Meadows, Aaron, McCarty, Douglas M., Fu, Haiyan
Publicado 2013

Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery

por Fu, Haiyan, Cataldi, Marcela P, Ware, Tierra A, Zaraspe, Kimberly, Meadows, Aaron S, Murrey, Darren A, McCarty, Douglas M
Publicado 2016

Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice

por McCarty, Douglas M., DiRosario, Julianne, Gulaid, Kadra, Killedar, Smruti, Oosterhof, Arie, van Kuppevelt, Toin H., Martin, Paul T., Fu, Haiyan
Publicado 2011

Production of rAAV by plasmid transfection induces antiviral and inflammatory responses in suspension HEK293 cells

por Chung, Cheng-Han, Murphy, Christopher M., Wingate, Vincent P., Pavlicek, Jeffrey W., Nakashima, Reiko, Wei, Wei, McCarty, Douglas, Rabinowitz, Joseph, Barton, Erik
Publicado 2023

Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats

por Storek, Benjamin, Harder, Nina M, Banck, Michaela S, Wang, Cheng, McCarty, Douglas M, Janssen, William GM, Morrison, John H, Walsh, Christopher E, Beutler, Andreas S
Publicado 2006

Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

por Sabatino, Denise E., Bushman, Frederic D., Chandler, Randy J., Crystal, Ronald G., Davidson, Beverly L., Dolmetsch, Ricardo, Eggan, Kevin C., Gao, Guangping, Gil-Farina, Irene, Kay, Mark A., McCarty, Douglas M., Montini, Eugenio, Ndu, Adora, Yuan, Jing
Publicado 2022

Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles

por Palazzi, Xavier, Pardo, Ingrid D., Sirivelu, Madhu P., Newman, Leah, Kumpf, Steven W., Qian, Jessie, Franks, Tania, Lopes, Sarah, Liu, June, Monarski, Laura, Casinghino, Sandra, Ritenour, Casey, Ritenour, Hayley, Dubois, Christopher, Olson, Jennifer, Graves, John, Alexander, Kristin E., Coskran, Timothy, Lanz, Thomas A., Brady, Joseph, McCarty, Douglas, Suryanarayan, Somanathan, Whiteley, Laurence O.
Publicado 2022