Patricia Dickson, M.D.

Publications

A selected list of publications:

Vite Charles H, Nestrasil Igor, Mlikotic Anton, Jens Jackie K, Snella Elizabeth M, Gross William, Shapiro Elsa G, Kovac Victor, Provenzale James M, Chen Steven, Le Steven Q, Kan Shih-hsin, Banakar Shida, Wang Raymond Y, Haskins Mark E, Ellinwood N Matthew, Dickson Patricia I   Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I Comparative medicine, 2013; 63(2): 163-73.
Vera Moin, Lester Thomas, Zhao Bin, Tiger Pascale, Clarke Scott, Tippin Brigette L, Passage Merry B, Le Steven Q, Femenia Javier, Lemontt Jeffrey F, Kakkis Emil D, Dickson Patricia I   Mannose 6-phosphate conjugation is not sufficient to allow induction of immune tolerance to phenylalanine ammonia-lyase in dogs JIMD reports, 2013; 8(2): 63-72.
Tippin Brigette L, Troitskaya Larisa, Kan Shih-hsin, Todd Amanda K, Le Steven Q, Dickson Patricia I   Biochemical characterization of fluorescent-labeled recombinant human alpha-L-iduronidase in vitro Biotechnology and applied biochemistry, 2012; 58(6): 391-6.
Dickson Patricia I, Ellinwood N Matthew, Brown Jillian R, Witt Robert G, Le Steven Q, Passage Merry B, Vera Moin U, Crawford Brett E   Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I Molecular genetics and metabolism, 2012; 106(1): 68-72.
Chen Agnes, Vogler Carole, McEntee Michael, Hanson Stephen, Ellinwood N Matthew, Jens Jackie, Snella Elizabeth, Passage Merry, Le Steven, Guerra Catalina, Dickson Patricia   Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase APMIS : acta pathologica, microbiologica, et immunologica Scandinavica, 2011; 119(8): 513-21.
Dickson Patricia I, Chen Agnes H   Intrathecal enzyme replacement therapy for mucopolysaccharidosis I: translating success in animal models to patients Current pharmaceutical biotechnology, 2011; 12(6): 946-55.
Dickson Patricia I, Hanson Stephen, McEntee Michael F, Vite Charles H, Vogler Carole A, Mlikotic Anton, Chen Agnes H, Ponder Katherine P, Haskins Mark E, Tippin Brigette L, Le Steven Q, Passage Merry B, Guerra Catalina, Dierenfeld Ashley, Jens Jackie, Snella Elizabeth, Kan Shih-Hsin, Ellinwood N Matthew   Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I Molecular genetics and metabolism, 2010; 101(2-3): 115-22.
Ezra Navid, Tetteh Beatrice, Diament Michael, Jonas Adam J, Dickson Patricia   Hereditary multiple exostoses with spine involvement in a 4-year-old boy American journal of medical genetics. Part A, 2010; 152A(5): 1264-7.
Chen Agnes, Dickson Patricia   Intrathecal enzyme replacement therapy to treat spinal cord compression in mucopolysaccharidosis: Overview and rationale Journal of pediatric rehabilitation medicine, 2010; 3(1): 7-11.
Dierenfeld AD, McEntee MF, Passage M, Le S, Jens JK, Snella EM, Kline KL, Parkes JD, Ware WA, Moran LE, Wengert JA, Whitley RD, Betts DM, Boal AM, Riedesel EA, Gross W, Ellinwood NM, Dickson PI   Replacing the enzyme α-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I, Science Translational Medicine, 2010; 2(60): ra89.
Passage M B, Krieger A W, Peinovich M C, Lester T, Le S Q, Dickson P I, Kakkis E D   Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs Journal of inherited metabolic disease, 2009; 110(3): .
Carlstrom Lucas P, Jens Jackie K, Dobyns Marley E, Passage Merry, Dickson Patricia I, Ellinwood N Matthew   Inadvertent propagation of factor VII deficiency in a canine mucopolysaccharidosis type I research breeding colony Comparative medicine, 2009; 59(4): 378-82.
Dickson Patricia, Peinovich Maryn, McEntee Michael, Lester Thomas, Le Steven, Krieger Aimee, Manuel Hayden, Jabagat Catherine, Passage Merry, Kakkis Emil D   Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I The Journal of clinical investigation, 2008; 118(8): 2868-76.
Munoz-Rojas Maria-Veronica, Vieira Taiane, Costa Ronaldo, Fagondes Simone, John Angela, Jardim Laura Bannach, Vedolin Leonardo M, Raymundo Marcia, Dickson Patricia I, Kakkis Emil, Giugliani Roberto   Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression American journal of medical genetics. Part A, 2008; 146A(19): 2538-44.
Dickson Patricia, McEntee Michael, Vogler Carole, Le Steven, Levy Beth, Peinovich Maryn, Hanson Stephen, Passage Merry, Kakkis Emil   Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid Molecular genetics and metabolism, 2007; 91(1): 61-8.
Belichenko P V, Dickson P I, Passage M, Jungles S, Mobley W C, Kakkis E D   Penetration, diffusion, and uptake of recombinant human alpha-L-iduronidase after intraventricular injection into the rat brain Molecular genetics and metabolism, 2005; 86(1-2): 141-9.
Dickson Patricia I, Briones Norman Y, Baylen Barry G, Jonas Adam J, French Samuel W, Lin Henry J   Costello syndrome with pancreatic islet cell hyperplasia American journal of medical genetics. Part A, 2004; 130A(4): 402-5.
Kakkis E, McEntee M, Vogler C, Le S, Levy B, Belichenko P, Mobley W, Dickson P, Hanson S, Passage M   Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I Molecular genetics and metabolism, 2004; 83(1-2): 163-74.
Joyce James J, Dickson Patricia I, Qi Ning, Noble Julie E, Raj J Usha, Baylen Barry G   Normal right and left ventricular mass development during early infancy The American journal of cardiology, 2004; 93(6): 797-801.
Dickson Patricia I   Delivering drugs to the central nervous system: an overview Drug delivery and translational research, 2012; 2(3): 145-51.
Choi Joshua, Dickson Patricia, Calabrese Evan, Chen Steven, White Leonard, Ellingwood Matthew, Provenzale James M   Predicting degree of myelination based on diffusion tensor imagining of canines with mucopolysaccharidosis type I The neuroradiology journal, 2015; 28(6): 562-73.
Dierenfeld Ashley D, McEntee Michael F, Vogler Carole A, Vite Charles H, Chen Agnes H, Passage Merry, Le Steven, Shah Sahil, Jens Jackie K, Snella Elizabeth M, Kline Karen L, Parkes Jennifer D, Ware Wendy A, Moran Lori E, Fales-Williams Amanda J, Wengert Jane A, Whitley R David, Betts Daniel M, Boal Amy M, Riedesel Elizabeth A, Gross William, Ellinwood N Matthew, Dickson Patricia I   Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I Science translational medicine, 2010; 2(60): 60ra89.
Lyons Jeremiah A, Dickson Patricia I, Wall Jonathan S, Passage Merry B, Ellinwood N Matthew, Kakkis Emil D, McEntee Michael F   Arterial pathology in canine mucopolysaccharidosis-I and response to therapy Laboratory investigation; a journal of technical methods and pathology, 2011; 91(5): 665-74.
Kishnani Priya S, Dickson Patricia I, Muldowney Laurie, Lee Jessica J, Rosenberg Amy, Abichandani Rekha, Bluestone Jeffrey A, Burton Barbara K, Dewey Maureen, Freitas Alexandra, Gavin Derek, Griebel Donna, Hogan Melissa, Holland Stephen, Tanpaiboon Pranoot, Turka Laurence A, Utz Jeanine J, Wang Yow-Ming, Whitley Chester B, Kazi Zoheb B, Pariser Anne R   Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction Molecular genetics and metabolism, 2015; 28(6): .
Provenzale James M, Nestrasil Igor, Chen Steven, Kan Shih-Hsin, Le Steven Q, Jens Jacqueline K, Snella Elizabeth M, Vondrak Kristen N, Yee Jennifer K, Vite Charles H, Elashoff David, Duan Lewei, Wang Raymond Y, Ellinwood N Matthew, Guzman Miguel A, Shapiro Elsa G, Dickson Patricia I   Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis I Experimental neurology, 2015; 273(6): 1-10.
Dickson Patricia I, Kaitila Ilkka, Harmatz Paul, Mlikotic Anton, Chen Agnes H, Victoroff Alla, Passage Merry B, Madden Jacqueline, Le Steven Q, Naylor David E, Naylor David E   Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I Molecular genetics and metabolism, 2015; 116(1-2): 69-74.
Maarup Timothy J, Chen Agnes H, Porter Forbes D, Farhat Nicole Y, Ory Daniel S, Sidhu Rohini, Jiang Xuntian, Dickson Patricia I   Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1 Molecular genetics and metabolism, 2015; 116(1-2): 75-9.
Dickson Patricia I, Tolar Jakub   The individual (single patient) IND for inborn errors of metabolism Molecular genetics and metabolism, 2015; 116(1-2): 1-3.
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   A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I Molecular therapy. Methods & clinical development, 2015; 2(1-2): 14068.
Shah Anuja, Miller Clinton J, Nast Cynthia C, Adams Mark D, Truitt Barbara, Tayek John A, Tong Lili, Mehtani Parag, Monteon Francisco, Sedor John R, Clinkenbeard Erica L, White Kenneth, Mehrotra Rajnish, LaPage Janine, Dickson Patricia, Adler Sharon G, Iyengar Sudha K   Severe vascular calcification and tumoral calcinosis in a family with hyperphosphatemia: a fibroblast growth factor 23 mutation identified by exome sequencing Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association – European Renal Association, 2014; 29(12): 2235-43.
Calias Pericles, Banks William A, Begley David, Scarpa Maurizio, Dickson Patricia   Intrathecal delivery of protein therapeutics to the brain: a critical reassessment Pharmacology & therapeutics, 2014; 144(2): 114-22.
Kan Shih-Hsin, Aoyagi-Scharber Mika, Le Steven Q, Vincelette Jon, Ohmi Kazuhiro, Bullens Sherry, Wendt Daniel J, Christianson Terri M, Tiger Pascale M N, Brown Jillian 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   Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB Proceedings of the National Academy of Sciences of the United States of America, 2014; 111(41): 14870-5.
Wang Raymond Y, Aminian Afshin, McEntee Michael F, Kan Shih-Hsin, Simonaro Calogera M, Lamanna William C, Lawrence Roger, Ellinwood N Matthew, Guerra Catalina, Le Steven Q, Dickson Patricia I, Esko Jeffrey D   Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I Molecular genetics and metabolism, 2014; 112(4): 286-93.
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   Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts The Biochemical journal, 2014; 458(2): 281-9.
Lawrence Roger, Brown Jillian R, Lorey Fred, Dickson Patricia I, Crawford Brett E, Esko Jeffrey D   Glycan-based biomarkers for mucopolysaccharidoses Molecular genetics and metabolism, 2014; 111(2): 73-83.
Vera Moin, Le Steven, Kan Shih-Hsin, Garban Hermes, Naylor David, Mlikotic Anton, Kaitila Ilkka, Harmatz Paul, Chen Agnes, Dickson Patricia   Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients Pediatric research, 2013; 74(6): 712-20.

Joseph Lasky III, M.D.

Publications

A selected list of publications:

Lasky Joseph L, Panosyan Eduard H, Plant Ashley, Davidson Tom, Yong William H, Prins Robert M, Liau Linda M, Moore Theodore B   Autologous tumor lysate-pulsed dendritic cell immunotherapy for pediatric patients with newly diagnosed or recurrent high-grade gliomas Anticancer research, 2013; 33(5): 2047-56.
Panosyan Eduard H, Ikeda Alan K, Chang Vivian Y, Laks Dan R, Reeb Charles L, Bowles La Vette, Lasky Joseph L, Moore Theodore B   High-dose chemotherapy with autologous hematopoietic stem-cell rescue for pediatric brain tumor patients: a single institution experience from UCLA Journal of transplantation, 2011; 2011(5): 740673.
Lasky Joseph L, Choe Meeryo, Nakano Ichiro   Cancer stem cells in pediatric brain tumors Current stem cell research & therapy, 2009; 4(4): 298-305.
Mehta Rupal I, Cutler Aaron R, Lasky Joseph L, Yong William H, Lerner Jason T, Hirota Brandon K, Salamon Noriko, Mathern Gary W, Vinters Harry V   “Primary” leptomeningeal medulloblastoma Human pathology, 2009; 40(11): 1661-5.
Guo Wei, Lasky Joseph L, Chang Chun-Ju, Mosessian Sherly, Lewis Xiaoman, Xiao Yun, Yeh Jennifer E, Chen James Y, Iruela-Arispe M Luisa, Varella-Garcia Marileila, Wu Hong   Multi-genetic events collaboratively contribute to Pten-null leukaemia stem-cell formation Nature, 2008; 453(7194): 529-33.
Ela-Dalman Noa, Arnold Anthony C, Chang Louis K, Velez Federico G, Lasky Joseph L   Abducens nerve ocular neuromyotonia following non-sellar or parasellar tumors Strabismus, 2008; 15(3): 149-51.
Lasky Joseph L, Choi Eun Jun, Johnston Samantha, Yong William H, Lazareff Jorge, Moore Theodore   Congenital brain tumors: case series and review of the literature Journal of pediatric hematology/oncology, 2008; 30(4): 326-31.
Lasky Joseph L, Liau Linda M   Targeting stem cells in brain tumors Technology in cancer research & treatment, 2006; 5(3): 251-60.
Luu Quang C, Lasky Joseph L, Moore Theodore B, Nelson Scott, Wang Marilene B   Treatment of embryonal rhabdomyosarcoma of the sinus and orbit with chemotherapy, radiation, and endoscopic surgery Journal of pediatric surgery, 2006; 41(6): e15-7.
Guo Wei, Lasky Joseph L, Wu Hong   Cancer stem cells Pediatric research, 2006; 59(4 Pt 2): 59R-64R.

Harley Kornblum, M.D., Ph.D.

Biography

Dr. Kornblum is a basic and translational researcher in the Intellectual and Developmental Disabilities Research Center of the Semel Research Institute. He is Professor of Psychiatry, Pharmacology and Pediatrics at the David Geffen School of Medicine at UCLA. He is also an attending Pediatric Neurologist at Mattel Children’s Hospital at UCLA and the founding director of the Neural Stem Cell Research Center. He is a member of the Brain Research Institute, The Broad Stem Cell Research Center and the Molecular Biology Institute and serves on the Steering Committe of the Broad Stem Cell Research Center. Dr. Kornblum received his AB degree from Washington University in St. Louis and his MD and PhD degrees from UC Irvine. He trained as a Pediatrics Resident and Pediatric Neurology Fellow at UCLA. He did undergraduate research with Dr. Eugene M. Johnson and received his PhD in the laboratory of Frances M. Leslie. He pursued postdoctoral research with Dr. Christine M. Gall at UCI and Dr. Harry Chugani at UCLA. Dr. Kornblum is a frequent lecturer on the topics of neural stem cells and brain tumors at conferences and universities world-wide and has published over 100 research articles and book chapters. Dr. Kornblum is the recipient of numerous grants from the Federal Government as well as philanthropic foundations. He served as the Eleanor Leslie Professor of Pioneering Brain Research.

Rebecca Stockton, Ph.D.

Biography

Dr. Stockton is a vascular biologist and specialist in endothelial cell signaling. She earned a PhD in Molecular and Cellular Biology from the University of Massachusetts Amherst, and trained as a postdoctoral fellow at the University of Virginia. She was Assistant Adjunct Professor at University of California San Diego, and established a lab at Harbor-UCLA Medical Center Dept of Pediatrics in 2012 as Assistant Professor of Pediatrics. She has extensive experience in the biology of vascular endothelial RhoGTPase signaling, conducting structure/function studies of in vitro and in vivo endothelial permeability and vascular barrier function, and assessing the basic mechanisms of vascular development and dysfunction in disease. Her work has been funded by NIH and the American Heart Association. She is a contributor to multiple groundbreaking studies defining aberrant endothelial signaling in Cerebral Cavernous Malformations (CCM) produced by mutations of the KRIT1, CCM2 and PDCD10 (CCM3) genes. As a junior faculty member at UC San Diego, her group reported the first endothelial expression and biological function regulating endothelial junctional stability of CCM gene product protein KRIT1. Ensuing collaborations with colleagues at University of Chicago and Duke University subsequently reported evidence for disrupted Rho/Rho-kinase signaling driving endothelial pathology producing brain lesions in this disease, identified a potential therapy, and reported in model mouse studies the first successful reduction of CCM brain lesion formation through a drug treatment. The Stockton Lab’s current studies focus on identifying additional protein signaling participants in multiple cerebrovascular pathologies.

Publications

A selected list of publications:

Shenkar Robert, Shi Changbin, Rebeiz Tania, Stockton Rebecca A, McDonald David A, Mikati Abdul Ghani, Zhang Lingjiao, Austin Cecilia, Akers Amy L, Gallione Carol J, Rorrer Autumn, Gunel Murat, Min Wang, Marcondes de Souza Jorge, Lee Connie, Marchuk Douglas A, Awad Issam A   Exceptional aggressiveness of cerebral cavernous malformation disease associated with PDCD10 mutations Genetics in medicine : official journal of the American College of Medical Genetics, 2015; 17(3): 188-96.
McDonald David A, Shi Changbin, Shenkar Robert, Stockton Rebecca A, Liu Feifei, Ginsberg Mark H, Marchuk Douglas A, Awad Issam A   Fasudil decreases lesion burden in a murine model of cerebral cavernous malformation disease Stroke; a journal of cerebral circulation, 2012; 43(2): 571-4.
Liu Jian J, Stockton Rebecca A, Gingras Alexandre R, Ablooglu Ararat J, Han Jaewon, Bobkov Andrey A, Ginsberg Mark H   A mechanism of Rap1-induced stabilization of endothelial cell–cell junctions Molecular biology of the cell, 2011; 22(14): 2509-19.
McDonald David A, Shenkar Robert, Shi Changbin, Stockton Rebecca A, Akers Amy L, Kucherlapati Melanie H, Kucherlapati Raju, Brainer James, Ginsberg Mark H, Awad Issam A, Marchuk Douglas A   A novel mouse model of cerebral cavernous malformations based on the two-hit mutation hypothesis recapitulates the human disease Human molecular genetics, 2011; 20(2): 211-22.
Stockton Rebecca A, Shenkar Robert, Awad Issam A, Ginsberg Mark H   Cerebral cavernous malformations proteins inhibit Rho kinase to stabilize vascular integrity The Journal of experimental medicine, 2010; 207(4): 881-96.
Goldfinger Lawrence E, Tzima Eleni, Stockton Rebecca, Kiosses William B, Kinbara Kayoko, Tkachenko Eugene, Gutierrez Edgar, Groisman Alex, Nguyen Phu, Chien Shu, Ginsberg Mark H   Localized alpha4 integrin phosphorylation directs shear stress-induced endothelial cell alignment Circulation research, 2008; 103(2): 177-85.
Jones Christopher A, London Nyall R, Chen Haoyu, Park Kye Won, Sauvaget Dominique, Stockton Rebecca A, Wythe Joshua D, Suh Wonhee, Larrieu-Lahargue Frederic, Mukouyama Yoh-Suke, Lindblom Per, Seth Pankaj, Frias Antonio, Nishiya Naoyuki, Ginsberg Mark H, Gerhardt Holger, Zhang Kang, Li Dean Y   Robo4 stabilizes the vascular network by inhibiting pathologic angiogenesis and endothelial hyperpermeability Nature medicine, 2008; 14(4): 448-53.
Glading Angela, Han Jaewon, Stockton Rebecca A, Ginsberg Mark H   KRIT-1/CCM1 is a Rap1 effector that regulates endothelial cell cell junctions The Journal of cell biology, 2007; 179(2): 247-54.
Stockton Rebecca, Reutershan Jörg, Scott David, Sanders John, Ley Klaus, Schwartz Martin Alexander   Induction of vascular permeability: beta PIX and GIT1 scaffold the activation of extracellular signal-regulated kinase by PAK Molecular biology of the cell, 2007; 18(6): 2346-55.
Reutershan Jörg, Stockton Rebecca, Zarbock Alexander, Sullivan Gail W, Chang Daniel, Scott David, Schwartz Martin A, Ley Klaus   Blocking p21-activated kinase reduces lipopolysaccharide-induced acute lung injury by preventing polymorphonuclear leukocyte infiltration American journal of respiratory and critical care medicine, 2007; 175(10): 1027-35.
Orr A Wayne, Stockton Rebecca, Simmers Michael B, Sanders John M, Sarembock Ian J, Blackman Brett R, Schwartz Martin Alexander   Matrix-specific p21-activated kinase activation regulates vascular permeability in atherogenesis The Journal of cell biology, 2007; 176(5): 719-27.
Tsai Irene Y, Kimura Masahiro, Stockton Rebecca, Green J Angelo, Puig Ricardo, Jacobson Bruce, Russell Thomas P   Fibroblast adhesion to micro- and nano-heterogeneous topography using diblock copolymers and homopolymers Journal of biomedical materials research. Part A, 2004; 71(3): 462-9.
Stockton Rebecca A, Schaefer Erik, Schwartz Martin Alexander   p21-activated kinase regulates endothelial permeability through modulation of contractility The Journal of biological chemistry, 2004; 279(45): 46621-30.
Green J Angelo, Stockton Rebecca A, Johnson Christopher, Jacobson Bruce S   5-lipoxygenase and cyclooxygenase regulate wound closure in NIH/3T3 fibroblast monolayers American journal of physiology. Cell physiology, 2004; 287(2): C373-83.