Treatment of brain disease in the mucopolysaccharidoses von Scarpa, Maurizio, Orchard, Paul J., Schulz, Angela, Dickson, Patricia I., Haskins, Mark E., Escolar, Maria L., Giugliani, Roberto

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

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Phenotypic expansion of KMT2D‐related disorder: Beyond Kabuki syndrome von Baldridge, Dustin, Spillmann, Rebecca C., Wegner, Daniel J., Wambach, Jennifer A., White, Frances V., Sisco, Kathleen, Toler, Tomi L., Dickson, Patricia I., Cole, F. Sessions, Shashi, Vandana, Grange, Dorothy K.

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The Epstein-Barr Virus Glycoprotein gp150 Forms an Immune-Evasive Glycan Shield at the Surface of Infected Cells von Gram, Anna M, Oosenbrug, Timo, Lindenbergh, Marthe F S, Büll, Christian, Comvalius, Anouskha, Dickson, Kathryn J I, Wiegant, Joop, Vrolijk, Hans, Lebbink, Robert Jan, Wolterbeek, Ron, Adema, Gosse J, Griffioen, Marieke, Heemskerk, Mirjam H M, Tscharke, David C, Hutt-Fletcher, Lindsey M, Wiertz, Emmanuel J H J, Hoeben, Rob C, Ressing, Maaike E

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Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB von Kan, Shih-hsin, Aoyagi-Scharber, Mika, Le, Steven Q., Vincelette, Jon, Ohmi, Kazuhiro, Bullens, Sherry, Wendt, Daniel J., Christiansen, Terri M., Tiger, Pascale M. N., Brown, Julian R., Lawrence, Roger, Yip, Bryan K., Holtzinger, John, Bagri, Anil, Crippen-Harmon, Danielle, Vondrak, Kristen N., Chen, Zhi, Hague, Chuck M., Woloszynek, Josh C., Cheung, Diana S., Webster, Katherine A., Adintori, Evan G., Lo, Melanie J., Wong, Wesley, Fitzpatrick, Paul A., LeBowitz, Jonathan H., Crawford, Brett E., Bunting, Stuart, Dickson, Patricia I., Neufeld, Elizabeth F.

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Immune-Mediated Inflammation May Contribute to the Pathogenesis of Cardiovascular Disease in Mucopolysaccharidosis Type I von Khalid, Omar, Vera, Moin U, Gordts, Philip L, Ellinwood, N Matthew, Schwartz, Philip H, Dickson, Patricia I, Esko, Jeffrey D, Wang, Raymond Y

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Genetics is Scary von Dickson, Patricia I

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Combining angiotensin receptor blockade and enzyme replacement therapy for vascular disease in mucopolysaccharidosis type I von Hurt, Sarah C., Vera, Moin U., Le, Steven Q., Kan, Shih-hsin, Bui, Quang, Dickson, Patricia I.

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Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts von Kan, Shih-hsin, Troitskaya, Larisa A, Sinow, Carolyn S, Haitz, Karyn, Todd, Amanda K, Di Stefano, Ariana, Le, Steven Q, Dickson, Patricia I, Tippin, Brigette L

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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 von 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.

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Postmortem diagnosis of very long chain acyl‐CoA dehydrogenase (VLCAD) deficiency in a neonate with sudden cardiac death von Singh, Prapti, Amaro, Deirdre, Obi, Olugbemisola, Kiran, FNU, Hediger, Erin, Toler, Tomi L., Dickson, Patricia I., Grange, Dorothy K.

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

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ONSHORE SEISMIC IMAGING USING DIFFERENT DATA DOMAIN IN NORTHERN NIGER DELTA, NIGERIA von I. Dickson, Addah, I., Tamunobereton-ari, I. Opiriyabo, Horsfall

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Fatal COVID‐19 infection in a patient with long‐chain 3‐hydroxyacyl‐CoA dehydrogenase deficiency: A case report von Wongkittichote, Parith, Watson, James R., Leonard, Jennifer M., Toolan, Elizabeth R., Dickson, Patricia I., Grange, Dorothy K.

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Evaluation of artificial signal peptides for secretion of two lysosomal enzymes in CHO cells von Cheng, Kai-Wen, Wang, Feng, Lopez, George A, Singamsetty, Srikanth, Wood, Jill, Dickson, Patricia I, Chou, Tsui-Fen

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Mucopolysaccharidoses type I gene therapy von Hurt, Sarah C., Dickson, Patricia I., Curiel, David T.

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Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice von Clarke, Don, Pearse, Yewande, Kan, Shih-hsin, Le, Steven Q., Sanghez, Valentina, Cooper, Jonathan D., Dickson, Patricia I., Iacovino, Michelina

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D-mannose as a new therapy for fucokinase deficiency-related congenital disorder of glycosylation (FCSK-CDG) von Starosta, Rodrigo Tzovenos, Lee, Angela J., Toolan, Elizabeth R., He, Miao, Wongkittichote, Parith, Daniel, Earnest James Paul, Radenkovic, Silvia, Budhraja, Rohit, Pandey, Akhilesh, Sharma, Jaiprakash, Morava, Eva, Nguyen, Hoanh, Dickson, Patricia I.

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Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy von Holley, Rebecca J, Ellison, Stuart M, Fil, Daniel, O'Leary, Claire, McDermott, John, Senthivel, Nishanthi, Langford-Smith, Alexander W W, Wilkinson, Fiona L, D'Souza, Zelpha, Parker, Helen, Liao, Aiyin, Rowlston, Samuel, Gleitz, Hélène F E, Kan, Shih-Hsin, Dickson, Patricia I, Bigger, Brian W

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Diagnostic yield of exome sequencing in congenital vertical talus von Tayebi, Naeimeh, Charng, Wu-Lin, Dickson, Patricia I., Dobbs, Matthew B., Gurnett, Christina A.

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