Mucopolysaccharidoses type I gene therapy

por Hurt, Sarah C., Dickson, Patricia I., Curiel, David T.
Publicado 2021

Fatal COVID‐19 infection in a patient with long‐chain 3‐hydroxyacyl‐CoA dehydrogenase deficiency: A case report

por Wongkittichote, Parith, Watson, James R., Leonard, Jennifer M., Toolan, Elizabeth R., Dickson, Patricia I., Grange, Dorothy K.
Publicado 2020

Postmortem diagnosis of very long chain acyl‐CoA dehydrogenase (VLCAD) deficiency in a neonate with sudden cardiac death

por Singh, Prapti, Amaro, Deirdre, Obi, Olugbemisola, Kiran, FNU, Hediger, Erin, Toler, Tomi L., Dickson, Patricia I., Grange, Dorothy K.
Publicado 2023

Elevated cerebral spinal fluid biomarkers in children with mucopolysaccharidosis I-H

por Raymond, Gerald V., Pasquali, Marzia, Polgreen, Lynda E., Dickson, Patricia I., Miller, Weston P., Orchard, Paul J., Lund, Troy C.
Publicado 2016

Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice

por Clarke, Don, Pearse, Yewande, Kan, Shih-hsin, Le, Steven Q., Sanghez, Valentina, Cooper, Jonathan D., Dickson, Patricia I., Iacovino, Michelina
Publicado 2018

Whole-Genome and Long-Read Sequencing Identify a Novel Mechanism in RFC1 Resulting in CANVAS Syndrome

por King, Katherine Abell, Wegner, Daniel J., Bucelli, Robert C., Shapiro, Jessica, Paul, Alexander J., Dickson, Patricia I., Wambach, Jennifer A.
Publicado 2022

Immune-Mediated Inflammation May Contribute to the Pathogenesis of Cardiovascular Disease in Mucopolysaccharidosis Type I

por Khalid, Omar, Vera, Moin U., Gordts, Philip L., Ellinwood, N. Matthew, Schwartz, Philip H., Dickson, Patricia I., Esko, Jeffrey D., Wang, Raymond Y.
Publicado 2016

Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease

por Pearse, Yewande, Clarke, Don, Kan, Shih-hsin, Le, Steven Q., Sanghez, Valentina, Luzzi, Anna, Pham, Ivy, Nih, Lina R., Cooper, Jonathan D., Dickson, Patricia I., Iacovino, Michelina
Publicado 2022

A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I

por Le, Steven Q., Kan, Shih-hsin, Clarke, Don, Sanghez, Valentina, Egeland, Martin, Vondrak, Kristen N., Doherty, Terence M., Vera, Moin U., Iacovino, Michelina, Cooper, Jonathan D., Sands, Mark S., Dickson, Patricia I.
Publicado 2017

A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-β-cyclodextrin for acute liver disease in infants with Niemann-Pick C1

por Reynolds, Margaret, Linneman, Laura A., Luna, Sofia, Warner, Barbara B., Turmelle, Yumirle P., Kulkarni, Sakil S., Jiang, Xuntian, Khanna, Geetika, Shinawi, Marwan, Porter, Forbes D., Ory, Daniel S., Cole, F. Sessions, Dickson, Patricia I.
Publicado 2021

Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes

por Eisengart, Julie B., Pierpont, Elizabeth I., Kaizer, Alexander M., Rudser, Kyle D., King, Kelly E., Pasquali, Marzia, Polgreen, Lynda E., Dickson, Patricia I., Le, Steven Q., Miller, Weston P., Tolar, Jakub, Orchard, Paul J., Lund, Troy C.
Publicado 2019

A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I

por Mendez, Daniel C, Stover, Alexander E, Rangel, Anthony D, Brick, David J, Nethercott, Hubert E, Torres, Marissa A, Khalid, Omar, Wong, Andrew MS, Cooper, Jonathan D, Jester, James V, Monuki, Edwin S, McGuire, Cian, Le, Steven Q, Kan, Shih-hsin, Dickson, Patricia I, Schwartz, Philip H
Publicado 2015

Impaired mitophagy in Sanfilippo a mice causes hypertriglyceridemia and brown adipose tissue activation

por Tillo, Miguel, Lamanna, William C., Dwyer, Chrissa A., Sandoval, Daniel R., Pessentheiner, Ariane R., Al-Azzam, Norah, Sarrazin, Stéphane, Gonzales, Jon C., Kan, Shih-Hsin, Andreyev, Alexander Y., Schultheis, Nicholas, Thacker, Bryan E., Glass, Charles A., Dickson, Patricia I., Wang, Raymond Y., Selleck, Scott B., Esko, Jeffrey D., Gordts, Philip L.S.M.
Publicado 2022

Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep

por Nelvagal, Hemanth R., Eaton, Samantha L., Wang, Sophie H., Eultgen, Elizabeth M., Takahashi, Keigo, Le, Steven Q., Nesbitt, Rachel, Dearborn, Joshua T., Siano, Nicholas, Puhl, Ana C., Dickson, Patricia I., Thompson, Gerard, Murdoch, Fraser, Brennan, Paul M., Gray, Mark, Greenhalgh, Stephen N., Tennant, Peter, Gregson, Rachael, Clutton, Eddie, Nixon, James, Proudfoot, Chris, Guido, Stefano, Lillico, Simon G., Whitelaw, C. Bruce A., Lu, Jui-Yun, Hofmann, Sandra L., Ekins, Sean, Sands, Mark S., Wishart, Thomas M., Cooper, Jonathan D.
Publicado 2022