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2. Immunotherapy targeting plasma ASM is protective in a mouse model of Alzheimer’s disease

Author

Choi, Byung Jo, Park, Min Hee, Park, Kang Ho, Han, Wan Hui, Yoon, Hee Ji, Jung, Hye Yoon, Hong, Ju Yeon, Chowdhury, Md Riad, Kim, Kyung Yeol, Lee, Jihoon, Song, Im-Sook, Pang, Minyeong, Choi, Min-Koo, Gulbins, Erich, Reichel, Martin, Kornhuber, Johannes, Hong, Chang-Won, Kim, Changho, Kim, Seung Hyun, Schuchman, Edward H., Jin, Hee Kyung, and Bae, Jae-sung

Published
2023
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4. Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A

Author

Samaranch, Lluis, Pérez-Cañamás, Azucena, Soto-Huelin, Beatriz, Sudhakar, Vivek, Jurado-Arjona, Jerónimo, Hadaczek, Piotr, Ávila, Jesús, Bringas, John R, Casas, Josefina, Chen, Haifeng, He, Xingxuan, Schuchman, Edward H, Cheng, Seng H, Forsayeth, John, Bankiewicz, Krystof S, and Ledesma, María Dolores

Subjects
Medical Biotechnology, Biomedical and Clinical Sciences, Gene Therapy, Rare Diseases, Biotechnology, Brain Disorders, Neurosciences, Neurodegenerative, Genetics, Neurological, Animals, Brain, Dependovirus, Genetic Therapy, Humans, Inflammation, Injections, Liver, Mice, Knockout, Motor Activity, Niemann-Pick Disease, Type A, Primates, Serogroup, Sphingomyelin Phosphodiesterase, Transgenes, Biological Sciences, Medical and Health Sciences, Medical biotechnology, Biomedical engineering
Abstract

Niemann-Pick disease type A (NPD-A) is a lysosomal storage disorder characterized by neurodegeneration and early death. It is caused by loss-of-function mutations in the gene encoding for acid sphingomyelinase (ASM), which hydrolyzes sphingomyelin into ceramide. Here, we evaluated the safety of cerebellomedullary (CM) cistern injection of adeno-associated viral vector serotype 9 encoding human ASM (AAV9-hASM) in nonhuman primates (NHP). We also evaluated its therapeutic benefit in a mouse model of the disease (ASM-KO mice). We found that CM injection in NHP resulted in widespread transgene expression within brain and spinal cord cells without signs of toxicity. CM injection in the ASM-KO mouse model resulted in hASM expression in cerebrospinal fluid and in different brain areas without triggering an inflammatory response. In contrast, direct cerebellar injection of AAV9-hASM triggered immune response. We also identified a minimally effective therapeutic dose for CM injection of AAV9-hASM in mice. Two months after administration, the treatment prevented motor and memory impairment, sphingomyelin (SM) accumulation, lysosomal enlargement, and neuronal death in ASM-KO mice. ASM activity was also detected in plasma from AAV9-hASM CM-injected ASM-KO mice, along with reduced SM amount and decreased inflammation in the liver. Our results support CM injection for future AAV9-based clinical trials in NPD-A as well as other lysosomal storage brain disorders.

Published
2019

8. Pentosan Polysulfate: Oral Versus Subcutaneous Injection in Mucopolysaccharidosis Type I Dogs

Author

Simonaro, Calogera M, Tomatsu, Shunji, Sikora, Tracy, Kubaski, Francyne, Frohbergh, Michael, Guevara, Johana M, Wang, Raymond Y, Vera, Moin, Kang, Jennifer L, Smith, Lachlan J, Schuchman, Edward H, and Haskins, Mark E

Subjects
Veterinary Sciences, Agricultural, Veterinary and Food Sciences, Biomedical and Clinical Sciences, Orphan Drug, Digestive Diseases, Brain Disorders, Mucopolysaccharidoses (MPS), Rare Diseases, Liver Disease, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Administration, Oral, Animals, Biomarkers, Blood Vessels, Cervical Vertebrae, Dogs, Female, Glycosaminoglycans, Humans, Injections, Subcutaneous, Male, Mucopolysaccharidosis I, Pentosan Sulfuric Polyester, Rats, Safety, General Science & Technology
Abstract

BackgroundWe previously demonstrated the therapeutic benefits of pentosan polysulfate (PPS) in a rat model of mucopolysaccharidosis (MPS) type VI. Reduction of inflammation, reduction of glycosaminoglycan (GAG) storage, and improvement in the skeletal phenotype were shown. Herein, we evaluate the long-term safety and therapeutic effects of PPS in a large animal model of a different MPS type, MPS I dogs. We focused on the arterial phenotype since this is one of the most consistent and clinically significant features of the model.Methodology/principal findingsMPS I dogs were treated with daily oral or biweekly subcutaneous (subQ) PPS at a human equivalent dose of 1.6 mg/kg for 17 and 12 months, respectively. Safety parameters were assessed at 6 months and at the end of the study. Following treatment, cytokine and GAG levels were determined in fluids and tissues. Assessments of the aorta and carotid arteries also were performed. No drug-related increases in liver enzymes, coagulation factors, or other adverse effects were observed. Significantly reduced IL-8 and TNF-alpha were found in urine and cerebrospinal fluid (CSF). GAG reduction was observed in urine and tissues. Increases in the luminal openings and reduction of the intimal media thickening occurred in the carotids and aortas of PPS-treated animals, along with a reduction of storage vacuoles. These results were correlated with a reduction of GAG storage, reduction of clusterin 1 staining, and improved elastin integrity. No significant changes in the spines of the treated animals were observed.ConclusionsPPS treatment led to reductions of pro-inflammatory cytokines and GAG storage in urine and tissues of MPS I dogs, which were most evident after subQ administration. SubQ administration also led to significant cytokine reductions in the CSF. Both treatment groups exhibited markedly reduced carotid and aortic inflammation, increased vessel integrity, and improved histopathology. We conclude that PPS may be a safe and useful therapy for MPS I, either as an adjunct or as a stand-alone treatment that reduces inflammation and GAG storage.

Published
2016

11. Control of CD1d-restricted antigen presentation and inflammation by sphingomyelin

Author

Melum, Espen, Jiang, Xiaojun, Baker, Kristi D., Macedo, M. Fatima, Fritsch, Jürgen, Dowds, C. Marie, Wang, Jing, Pharo, Anne, Kaser, Arthur, Tan, Corey, Pereira, Catia S., Kelly, Samuel L., Duan, Jingjing, Karlsen, Tom H., Exley, Mark A., Schütze, Stefan, Zajonc, Dirk M., Merrill, Alfred H., Schuchman, Edward H., Zeissig, Sebastian, and Blumberg, Richard S.

Published
2019
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13. List of contributors

Author

Ah Mew, Nicholas, primary, Akamatsu, Wado, additional, Akman, Hasan Orhan, additional, AlHakeem, Afnan, additional, Aoyama, Koji, additional, Artuch, Rafael, additional, Beck, Michael, additional, Bennett, C. Frank, additional, Berry, Gerard T., additional, Bissell, D. Montgomery, additional, Canine, Brenda, additional, Caskey, C. Thomas, additional, Casy, Widler, additional, Chinnery, Patrick F., additional, Chuang, David T., additional, Cook, Emily K., additional, Cox, Rody P., additional, De Jager, Philip L., additional, Demirbas, Didem, additional, Desnick, Robert J., additional, DiMauro, Salvatore, additional, Eichler, Florian S., additional, Elger, Bernice, additional, Emmanuele, Valentina, additional, Evans, Patricia, additional, Fogel, Brent L., additional, García-Cazorla, Àngels, additional, Gellera, Cinzia, additional, Golla, Sailaja, additional, Goodspeed, Kimberly, additional, Gospe, Sidney M., additional, Gray, Steven J., additional, Gropman, Andrea L., additional, Gu, Yian, additional, Guerrini, Renzo, additional, Gunn, Teresa M., additional, Hadziahmetovic, Una, additional, Haffner, Darrah, additional, Hagerman, R.J., additional, Harel, Tamar, additional, Head, Elizabeth, additional, Horvath, Rita, additional, Hosoi, Yasushi, additional, Hou, Ying-Chen Claire, additional, Hsiao, Jane, additional, Ishiura, Hiroyuki, additional, Jack, Clifford R., additional, Jakkamsetti, Vikram, additional, Johnson†, William G., additional, Jotterand, Fabrice, additional, Kane, John P., additional, Khorkova, Olga, additional, Kinoshita, Chisato, additional, Klompe, Sanne E., additional, Koehl, Lisa M., additional, Kruer, Michael C., additional, Kukull, Walter A., additional, Lane, Roger M., additional, Lee, Joseph H., additional, Leigh, M.J., additional, Ling, Qinglan, additional, Lupski, James R., additional, Luzi, Paola, additional, Ma, Qian, additional, Maegawa, Gustavo H.B., additional, Malloy, Mary J., additional, Margolis, Seth S., additional, Marin-Valencia, Isaac, additional, Mastrianni, James A., additional, Matalon, Dena, additional, Matalon, Reuben, additional, Matalon Rd, Kimberlee Michals, additional, Mathews, Jennifer M., additional, Mayeux, Richard, additional, McCurdy, Jennifer, additional, Meltzer, Meira R., additional, Menkes†, John H., additional, Miron, Justin, additional, Mitsui, Jun, additional, Miyajima, Hiroaki, additional, Monteggia, Lisa M., additional, Morris, Mary Ann, additional, Moser†, Hugo W., additional, Murray, Melissa E., additional, Nakaki, Toshio, additional, Nilsson, Nathalie, additional, Nishino, Ichizo, additional, Nordlie, Sandra M.H., additional, Nussbaum, Robert L., additional, Nyhan, William L., additional, Okano, Hideyuki, additional, Padilla-Lopez, Sergio, additional, Parrini, Elena, additional, Pascual, Juan M., additional, Pastores, Gregory M., additional, Patel, Shailendra B., additional, Patterson, Marc C., additional, Pena, Izabella A., additional, Picard, Cynthia, additional, Poirier, Judes, additional, Posey, Jennifer E., additional, Raymond, Gerald V., additional, Renthal, William, additional, Rosenblatt, David S., additional, Rossignol, Francis, additional, Salen, Gerald, additional, Sandhoff, Konrad, additional, Schiffmann, Raphael, additional, Schindler, Detlev, additional, Schmitt, Frederick A., additional, Schneider, Susanne A., additional, Schon, Eric A., additional, Schuchman, Edward H., additional, Seashore, Margretta Reed, additional, Shaffo, Frances C., additional, Shevell, Michael, additional, Sinnett, Sarah E., additional, Srour, Myriam, additional, Sternberg, Samuel H., additional, Sugie, Kazuma, additional, Szabla, Kristen L., additional, Taroni, Franco, additional, Tedeschi Dauar, Marina, additional, Tsuji, Shoji, additional, Uhlmann, Wendy R., additional, van Karnebeek, Clara, additional, Van Pelt, Kathryn L., additional, Vemuri, Prashanthi, additional, Venditti, Charles P., additional, Wahlestedt, Claes, additional, Wang, Bruce, additional, Watkins, David, additional, Wenger, David A., additional, Williams, Charles A., additional, Wilson, Golder N., additional, Wolf, Barry, additional, Wynn, R. Max, additional, and Yu, Hung-Chun, additional

Published
2020
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15. Modulation of Dietary Choline Uptake in a Mouse Model of Acid Sphingomyelinase Deficiency

Author

Gaudioso, Ángel, Moreno-Huguet, Pilar, Casas, Josefina, Schuchman, Edward H, and Ledesma, María Dolores

Subjects
Sphingomyelin, Phospholipid, Acid sphingomyelinase deficiency, Lysosomal storage disorder, Lipidomic, Ensure healthy lives and promote well-being for all at all ages, Choline
Abstract

Acid sphingomyelinase deficiency (ASMD) is a lysosomal storage disorder caused by mutations in the gene-encoding acid sphingomyelinase (ASM). ASMD impacts peripheral organs in all patients, including the liver and spleen. The infantile and chronic neurovisceral forms of the disease also lead to neuroinflammation and neurodegeneration for which there is no effective treatment. Cellular accumulation of sphingomyelin (SM) is a pathological hallmark in all tissues. SM is the only sphingolipid comprised of a phosphocholine group linked to ceramide. Choline is an essential nutrient that must be obtained from the diet and its deficiency promotes fatty liver disease in a process dependent on ASM activity. We thus hypothesized that choline deprivation could reduce SM production and have beneficial effects in ASMD. Using acid sphingomyelinase knock-out (ASMko) mice, which mimic neurovisceral ASMD, we have assessed the safety of a choline-free diet and its effects on liver and brain pathological features such as altered sphingolipid and glycerophospholipid composition, inflammation and neurodegeneration. We found that the choline-free diet was safe in our experimental conditions and reduced activation of macrophages and microglia in the liver and brain, respectively. However, there was no significant impact on sphingolipid levels and neurodegeneration was not prevented, arguing against the potential of this nutritional strategy to assist in the management of neurovisceral ASMD patients., This work was funded by a fellowship of the Wylder Nation Foundation to A.G. and by grants from the Spanish Ministry of Science and Innovation (PID2020-112830RB-I00) (AEI/FEDER, UE) and the Next Generation EU-CSIC funds (NeuroAging) to M.D.L.

Published
2023

16. Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A

Author

Bu, Jie, Ashe, Karen M, Bringas, John, Marshall, John, Dodge, James C, Cabrera-Salazar, Mario A, Forsayeth, John, Schuchman, Edward H, Bankiewicz, Krystof S, Cheng, Seng H, Shihabuddin, Lamya S, and Passini, Marco A

Subjects
Medical Biotechnology, Biomedical and Clinical Sciences, Brain Disorders, Neurosciences, Animals, Brain, Dependovirus, Disease Models, Animal, Enzyme-Linked Immunosorbent Assay, Genetic Therapy, Genetic Vectors, Injections, Macaca fascicularis, Male, Mice, Mice, Knockout, Niemann-Pick Disease, Type A, Primates, Sphingomyelin Phosphodiesterase, Biological Sciences, Technology, Medical and Health Sciences, Biotechnology, Genetics, Clinical sciences, Medical biotechnology
Abstract

Niemann-Pick disease Type A (NPA) is a neuronopathic lysosomal storage disease (LSD) caused by the loss of acid sphingomyelinase (ASM). The goals of the current study are to ascertain the levels of human ASM that are efficacious in ASM knockout (ASMKO) mice, and determine whether these levels can be attained in non-human primates (NHPs) using a multiple parenchymal injection strategy. Intracranial injections of different doses of AAV1-hASM in ASMKO mice demonstrated that only a small amount of enzyme (10 mg hASM/g tissue was reached. In monkeys, injection of 12 tracts of AAV1-hASM resulted in efficacious levels of enzyme in broad regions of the brain that was aided, in part, by axonal transport of adeno-associated virus (AAV) and movement through the perivascular space. This study demonstrates that a combination cortical, subcortical, and cerebellar injection protocol could provide therapeutic levels of hASM to regions of the NHP brain that are highly affected in NPA patients. The information from this study might help design new AAV-mediated enzyme replacement protocols for NPA and other neuronopathic LSDs in future clinical trials.

Published
2012

24. List of Contributors

Author

Alwan, Sura, primary, Clarke, Angus John, additional, Astrin, Kenneth H., additional, Bartsakoulia, Marina, additional, Burkardt, Deepika D’Cunha, additional, Chalikiopoulou, Constantina, additional, Deignan, Joshua L., additional, Desnick, Robert J., additional, Dhar, Shweta U., additional, Doyle, Debra Lochner, additional, Ferguson-Smith, Malcolm A., additional, Friedman, Jan M., additional, Graham Jr., John M., additional, Graziano, Daniel, additional, Grody, Wayne W., additional, Irving, Rachel, additional, Jones, Kenneth L., additional, Jones, Marilyn C., additional, Katsila, Theodora, additional, Khoury, Muin J., additional, McGinniss, Matthew J., additional, McGinniss, Molly A., additional, Moeschler, John B., additional, Panagiotara, Angeliki, additional, Patrinos, George P., additional, Scheuner, Maren T., additional, Schuchman, Edward H., additional, Sehgal, Amita, additional, Skoufas, Efthymios, additional, Spinner, Nancy B., additional, Trucco, Massimo, additional, Tsermpini, Evangelia-Eirini, additional, Williams, Marc S., additional, and Zhang, Shirley L., additional

Published
2019
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28. Contributors

Author

Abdel-Wahab, Omar, primary, Abrahm, Janet L., additional, Adams, Sharon, additional, Adewoye, Adeboye H., additional, Allen, Carl, additional, Ambinder, Richard F., additional, Anasetti, Claudio, additional, Anastasi, John, additional, Anderson, Julia A., additional, Antin, Joseph H., additional, Antony, Aśok C., additional, Araten, David J., additional, Armand, Philippe, additional, Armstrong, Gillian, additional, Armstrong, Scott A., additional, Arnold, Donald M., additional, Artz, Andrew S., additional, Awan, Farrukh T., additional, Baglin, Trevor P., additional, Benson, Don M., additional, Benz, Edward J., additional, Berliner, Nancy, additional, Bhagat, Govind, additional, Bhardwaj, Nina, additional, Bhatia, Ravi, additional, Bhatia, Smita, additional, Bhatt, Mihir D., additional, Bhatt, Vijaya Raj, additional, Bitan, Menachem, additional, Blinderman, Craig D., additional, Bollard, Catherine M., additional, Braun, Benjamin S., additional, Brenner, Malcolm K., additional, Brittenham, Gary M., additional, Brodsky, Robert A., additional, Brown, Myles, additional, Broxmeyer, Hal E., additional, Brummel-Ziedins, Kathleen, additional, Brunner, Andrew M., additional, Buadi, Francis K., additional, Burkhardt, Birgit, additional, Burns, Melissa, additional, Byrd, John C., additional, Caimi, Paolo F., additional, Caligiuri, Michael A., additional, Canavan, Michelle, additional, Cantor, Alan B., additional, Carcao, Manuel, additional, Carroll, Michael C., additional, Carty, Shannon A., additional, Castillo, Jorge J., additional, Chan, Anthony K.C., additional, Chapin, John, additional, Chiu, April, additional, Chute, John P., additional, Clark, David B., additional, Coates, Thomas D., additional, Cogle, Christopher R., additional, Connell, Nathan T., additional, Cooke, Elizabeth, additional, Cooley, Sarah, additional, Corradini, Paolo, additional, Creager, Mark A., additional, Creger, Richard J., additional, Cromwell, Caroline, additional, Crowther, Mark A., additional, Cushing, Melissa M., additional, Cutler, Corey, additional, Dang, Chi V., additional, Danial, Nika N., additional, Dave, Sandeep S., additional, DeCaprio, James A., additional, Dinauer, Mary C., additional, Dinner, Shira, additional, Diz-Küçükkaya, Reyhan, additional, Dodd, Roger Y., additional, Donato, Michele L., additional, Dorshkind, Kenneth, additional, Dotti, Gianpietro, additional, Dror, Yigal, additional, Dunleavy, Kieron, additional, Dvorak, Christopher C., additional, Ebert, Benjamin L., additional, Eck, Michael J., additional, Eikelboom, John W., additional, Epperla, Narendranath, additional, Ershler, William B., additional, Evans, William E., additional, Faderl, Stefan, additional, Ferrara, James L.M., additional, Filipovich, Alexandra Hult, additional, Fischer, Martin, additional, Fredenburgh, James C., additional, Friedman, Kenneth D., additional, Fuchs, Ephraim, additional, Fuller, Stephen J., additional, Gailani, David, additional, Galipeau, Jacques, additional, Gallagher, Patrick G., additional, Ganapathi, Karthik A., additional, Gardner, Lawrence B., additional, Gee, Adrian P., additional, Gerson, Stanton L., additional, Gertz, Morie A., additional, Giardina, Patricia J., additional, Gibson, Christopher J., additional, Golan, Karin, additional, Golub, Todd R., additional, Gonzales, Matthew J., additional, Gotlib, Jason, additional, Gottschalk, Stephen, additional, Grant, Marianne A., additional, Graubert, Timothy A., additional, Gregg, Xylina T., additional, Gribben, John G., additional, Gross, Dawn M., additional, Gruber, Tanja A., additional, Guitart, Joan, additional, Gurbuxani, Sandeep, additional, Gur-Cohen, Shiri, additional, Gutierrez, Alejandro, additional, Hamadani, Mehdi, additional, Hari, Parameswaran N., additional, Hartwig, John H., additional, Hayman, Suzanne R., additional, Hayward, Catherine P.M., additional, Hebbel, Robert P., additional, Heslop, Helen E., additional, Hillis, Christopher, additional, Hillyer, Christopher D., additional, Ho, Karin, additional, Hockenbery, David M., additional, Hoffman, Ronald, additional, Hogg, Kerstin E., additional, Holtan, Shernan G., additional, Horny, Hans-Peter, additional, Hsu, Yen-Michael S., additional, Hunter, Zachary R., additional, Huntington, James A., additional, Iancu-Rubin, Camelia, additional, Iqbal, Ali, additional, Isenman, David E., additional, Israels, Sara J., additional, Italiano, Joseph E., additional, Jaffe, Elaine S., additional, Jaffer, Iqbal H., additional, Jagannath, Sundar, additional, Jäger, Ulrich, additional, Jain, Nitin, additional, James, Paula, additional, Jeha, Sima, additional, Jordan, Michael B., additional, Josephson, Cassandra D., additional, Jung, Moonjung, additional, Kager, Leo, additional, Kambayashi, Taku, additional, Kanakry, Jennifer A., additional, Kantarjian, Hagop M., additional, Kaplan, Jason, additional, Karafin, Matthew S., additional, Karsan, Aly, additional, Kaufman, Randal J., additional, Kaufman, Richard M., additional, Keller, Frank G., additional, Kelly, Kara M., additional, Kessler, Craig M., additional, Key, Nigel S., additional, Keyzner, Alla, additional, Khandoga, Alexander G., additional, Khanna-Gupta, Arati, additional, Khatib-Massalha, Eman, additional, Klein, Harvey G., additional, Knoechel, Birgit, additional, Kollet, Orit, additional, Konkle, Barbara A., additional, Kontoyiannis, Dimitrios P., additional, Koreth, John, additional, Koretzky, Gary A., additional, Kotecha, Dipak, additional, Kremyanskaya, Marina, additional, Kumari, Anju, additional, Kuzel, Timothy M., additional, Küppers, Ralf, additional, Lacy, Martha Q., additional, Ladas, Elana, additional, Landier, Wendy, additional, Lapid, Kfir, additional, Lapidot, Tsvee, additional, Larson, Peter J., additional, Levi, Marcel, additional, Lewis, Russell E., additional, Liebman, Howard A., additional, Lillicrap, David, additional, Lim, Wendy, additional, Lin, Judith C., additional, Lindblad, Robert, additional, Lip, Gregory Y.H., additional, Little, Jane A., additional, Lohr, Jens G., additional, López, José A., additional, Luscinskas, Francis W., additional, Maciejewski, Jaroslaw P., additional, Majhail, Navneet S., additional, Manches, Olivier, additional, Mandle, Robert J., additional, Mann, Kenneth G., additional, Manno, Catherine S., additional, Marcogliese, Andrea N., additional, Mariani, Guglielmo, additional, Marincola, Francesco M., additional, Mascarenhas, John, additional, Massberg, Steffen, additional, McEver, Rodger P., additional, McGrath, Emer, additional, McKinney, Matthew S., additional, Mehta, Rohtesh S., additional, Mentzer, William C., additional, Merlini, Giampaolo, additional, Merryman, Reid, additional, Michel, Marc, additional, Migliaccio, Anna Rita, additional, Miller, Jeffrey S., additional, Mims, Martha P., additional, Mondoro, Traci Heath, additional, Moorehead, Paul, additional, Muniz, Luciana R., additional, Munshi, Nikhil C., additional, Najfeld, Vesna, additional, Nayak, Lalitha, additional, Nazy, Ishac, additional, Neff, Anne T., additional, Ness, Paul M., additional, Notarangelo, Luigi D., additional, O'Brien, Sarah H., additional, O'Connor, Owen A., additional, O'Donnell, Martin, additional, Olson, Amanda, additional, Orkin, Stuart H., additional, Pai, Menaka, additional, Pai, Sung-Yun, additional, Paidas, Michael, additional, Panch, Sandhya R., additional, Pande, Reena L., additional, Papayannopoulou, Thalia, additional, Parikh, Rahul, additional, Petersdorf, Effie W., additional, Peterson, Shane E., additional, Pittaluga, Stefania, additional, Ponce, Doris M., additional, Popolo, Laura, additional, Prchal, Josef T., additional, Pui, Ching-Hon, additional, Puigserver, Pere, additional, Rak, Janusz, additional, Ramos, Carlos A., additional, Rand, Jacob H., additional, Rand, Margaret L., additional, Rao, Dinesh S., additional, Ravandi, Farhad, additional, Rawlings, David J., additional, Reddy, Pavan, additional, Reding, Mark T., additional, Reiter, Andreas, additional, Rice, Lawrence, additional, Riese, Matthew J., additional, Ritchey, Arthur Kim, additional, Roberts, David J., additional, Roman, Elizabeth, additional, Rooney, Cliona M., additional, Rosen, Steven T., additional, Rosenthal, David S., additional, Rossmann, Marlies P., additional, Rot, Antal, additional, Rowley, Scott D., additional, Rubnitz, Jeffrey E., additional, Rydz, Natalia, additional, Salama, Mohamed E., additional, Sauk, Steven, additional, Saunthararajah, Yogen, additional, Savage, William, additional, Scadden, David, additional, Schaefer, Kristen G., additional, Schiffman, Fred, additional, Schneidewend, Robert, additional, Schrier, Stanley L., additional, Schuchman, Edward H., additional, Scullion, Bridget Fowler, additional, Selvaggi, Kathy J., additional, Senoo, Keitaro, additional, Shaheen, Montaser, additional, Shaz, Beth H., additional, Shelburne, Samuel A., additional, Shpall, Elizabeth J., additional, Shurin, Susan B., additional, Siegal, Deborah, additional, Silberstein, Leslie E., additional, Silberstein, Lev, additional, Silverstein, Roy L., additional, Sloan, Steven R., additional, Smith, Franklin O., additional, Smith, James W., additional, Smith, Katy, additional, Steensma, David P., additional, Steinberg, Martin H., additional, Stock, Wendy, additional, Storry, Jill R., additional, Stramer, Susan L., additional, Strauss, Ronald G., additional, Stroncek, David F., additional, Taylor, Justin, additional, Thota, Swapna, additional, Treon, Steven P., additional, Tulpule, Anil, additional, Valdes, Roberto Ferro, additional, Valent, Peter, additional, Vedantham, Suresh, additional, Vercellotti, Gregory M., additional, Verneris, Michael R., additional, Vichinsky, Elliott P., additional, von Andrian, Ulrich H., additional, Vose, Julie M., additional, Wagner, Andrew J., additional, Wang, Ena, additional, Wang, Jia-huai, additional, Warkentin, Theodore E., additional, Wasserstein, Melissa P., additional, Webster, Ann, additional, Weisdorf, Daniel J., additional, Weitz, Jeffrey I., additional, Westhoff, Connie M., additional, Wheeler, Allison P., additional, Widick, Page, additional, Wiley, James S., additional, William, Basem M., additional, Williams, David A., additional, Wilson, Wyndham H., additional, Wolfe, Joanne, additional, Wolgast, Lucia R., additional, Wood, Deborah, additional, Wu, Jennifer, additional, Yahalom, Joachim, additional, Yee, Donald L., additional, Younes, Anas, additional, Young, Neal S., additional, and Zeller, Michelle P., additional

Published
2018
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33. The clinical spectrum of SMA‐PME and in vitro normalization of its cellular ceramide profile

Author

Lee, Michelle M., primary, McDowell, Graeme S. V., additional, De Vivo, Darryl C., additional, Friedman, Daniel, additional, Berkovic, Samuel F., additional, Spanou, Maria, additional, Dinopoulos, Argirios, additional, Grand, Katheryn, additional, Sanchez‐Lara, Pedro A., additional, Allen‐Sharpley, Michelle, additional, Warman‐Chardon, Jodi, additional, Solyom, Alexander, additional, Levade, Thierry, additional, Schuchman, Edward H., additional, Bennett, Steffany A. L., additional, Dyment, David A., additional, and Pearson, Toni S., additional

Published
2022
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39. Discovery of a dual-action small molecule that improves neuropathological features of Alzheimer’s disease mice

Author

Park, Min Hee, primary, Park, Kang Ho, additional, Choi, Byung Jo, additional, Han, Wan Hui, additional, Yoon, Hee Ji, additional, Jung, Hye Yoon, additional, Lee, Jihoon, additional, Song, Im-Sook, additional, Lim, Dong Yu, additional, Choi, Min-Koo, additional, Lee, Yang-Ha, additional, Park, Cheol-Min, additional, Wang, Ming, additional, Jo, Jihoon, additional, Kim, Hee-Jin, additional, Kim, Seung Hyun, additional, Schuchman, Edward H., additional, Jin, Hee Kyung, additional, and Bae, Jae-sung, additional

Published
2022
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