Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model von Stoica, Lorelei, Todeasa, Sophia H., Cabrera, Gabriela Toro, Salameh, Johnny S., ElMallah, Mai K., Mueller, Christian, Brown Jr, Robert H., Sena-Esteves, Miguel

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Intracranial AAV‐IFN‐β gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model von GuhaSarkar, Dwijit, Neiswender, James, Su, Qin, Gao, Guangping, Sena‐Esteves, Miguel

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Spinal projections of the A5, A6 (locus coeruleus), and A7 noradrenergic cell groups in rats von Bruinstroop, Eveline, Cano, Georgina, Vanderhorst, Veronique G.J.M., Cavalcante, Judney C., Wirth, Jena, Sena-Esteves, Miguel, Saper, Clifford B.

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Mutation in protein disulfide isomerase A3 causes neurodevelopmental defects by disturbing endoplasmic reticulum proteostasis von Bilches Medinas, Danilo, Malik, Sajid, Yıldız‐Bölükbaşı, Esra, Borgonovo, Janina, Saaranen, Mirva J, Urra, Hery, Pulgar, Eduardo, Afzal, Muhammad, Contreras, Darwin, Wright, Madison T, Bodaleo, Felipe, Quiroz, Gabriel, Rozas, Pablo, Mumtaz, Sara, Díaz, Rodrigo, Rozas, Carlos, Cabral‐Miranda, Felipe, Piña, Ricardo, Valenzuela, Vicente, Uyan, Ozgun, Reardon, Christopher, Woehlbier, Ute, Brown, Robert H, Sena‐Esteves, Miguel, Gonzalez‐Billault, Christian, Morales, Bernardo, Plate, Lars, Ruddock, Lloyd W, Concha, Miguel L, Hetz, Claudio, Tolun, Aslıhan

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Life‐Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy von Johnson, Aime K., McCurdy, Victoria J., Gray‐Edwards, Heather L., Maguire, Anne S., Cochran, J. Nicholas, Gross, Amanda L., Skinner, Haleigh E., Randle, Ashley N., Shirley, Jamie L., Brunson, Brandon L., Bradbury, Allison M., Leroy, Stanley G., Hwang, Misako, Rockwell, Hannah E., Cox, Nancy R., Baker, Henry J., Seyfried, Thomas N., Sena‐Esteves, Miguel, Martin, Douglas R.

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Viral vectors for therapy of neurologic diseases von Choudhury, Sourav R., Hudry, Eloise, Maguire, Casey A., Sena-Esteves, Miguel, Breakefield, Xandra O., Grandi, Paola

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Gene Transfer to the CNS Using Recombinant Adeno‐Associated Virus von Stoica, Lorelei, Ahmed, Seemin S., Gao, Guangping, Sena‐Esteves, Miguel

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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system von Alterman, Julia F., Godinho, Bruno M. D. C., Hassler, Matthew R., Ferguson, Chantal M., Echeverria, Dimas, Sapp, Ellen, Haraszti, Reka A., Coles, Andrew H., Conroy, Faith, Miller, Rachael, Roux, Loic, Yan, Paul, Knox, Emily G., Turanov, Anton A., King, Robert M., Gernoux, Gwladys, Mueller, Christian, Gray-Edwards, Heather L., Moser, Richard P., Bishop, Nina C., Jaber, Samer M., Gounis, Matthew J., Sena-Esteves, Miguel, Pai, Athma A., DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

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Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation[S] von Allende, Maria L., Cook, Emily K., Larman, Bridget C., Nugent, Adrienne, Brady, Jacqueline M., Golebiowski, Diane, Sena-Esteves, Miguel, Tifft, Cynthia J., Proia, Richard L.

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Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System von Zhang, Hongwei, Yang, Bin, Mu, Xin, Ahmed, Seemin Seher, Su, Qin, He, Ran, Wang, Hongyan, Mueller, Christian, Sena-Esteves, Miguel, Brown, Robert, Xu, Zuoshang, Gao, Guangping

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Microvesicle-associated AAV Vector as a Novel Gene Delivery System von Maguire, Casey A, Balaj, Leonora, Sivaraman, Sarada, Crommentuijn, Matheus HW, Ericsson, Maria, Mincheva-Nilsson, Lucia, Baranov, Vladimir, Gianni, Davide, Tannous, Bakhos A, Sena-Esteves, Miguel, Breakefield, Xandra O, Skog, Johan

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Adeno Associated Viral Vector Delivered RNAi for Gene Therapy of SOD1 Amyotrophic Lateral Sclerosis von Stoica, Lorelei, Sena-Esteves, Miguel

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Brain-Derived Neurotrophic Factor Regulates Hedonic Feeding by Acting on the Mesolimbic Dopamine System von Cordeira, Joshua W, Frank, Lauren, Sena-Esteves, Miguel, Pothos, Emmanuel N, Rios, Maribel

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Bis(monoacylglycero)phosphate: a secondary storage lipid in the gangliosidoses von Akgoc, Zeynep, Sena-Esteves, Miguel, Martin, Douglas R., Han, Xianlin, d'Azzo, Alessandra, Seyfried, Thomas N.

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Selection of an Efficient AAV Vector for Robust CNS Transgene Expression von Hanlon, Killian S., Meltzer, Jonah C., Buzhdygan, Tetyana, Cheng, Ming J., Sena-Esteves, Miguel, Bennett, Rachel E., Sullivan, Timothy P., Razmpour, Roshanak, Gong, Yi, Ng, Carrie, Nammour, Josette, Maiz, Daniela, Dujardin, Simon, Ramirez, Servio H., Hudry, Eloise, Maguire, Casey A.

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Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease von Gray-Edwards, Heather L, Randle, Ashley N, Maitland, Stacy A, Benatti, Hector R, Hubbard, Spencer M, Canning, Peter F, Vogel, Matthew B, Brunson, Brandon L, Hwang, Misako, Ellis, Lauren E, Bradbury, Allison M, Gentry, Atoska S, Taylor, Amanda R, Wooldridge, Anne A, Wilhite, Dewey R, Winter, Randolph L, Whitlock, Brian K, Johnson, Jacob A, Holland, Merilee, Salibi, Nouha, Beyers, Ronald J, Sartin, James L, Denney, Thomas S, Cox, Nancy R, Sena-Esteves, Miguel, Martin, Douglas R

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Expression of ALS-PFN1 impairs vesicular degradation in iPSC-derived microglia von Funes, Salome, Jung, Jonathan, Gadd, Del Hayden, Mosqueda, Michelle, Zhong, Jianjun, Shankaracharya, Unger, Matthew, Stallworth, Karly, Cameron, Debra, Rotunno, Melissa S., Dawes, Pepper, Fowler-Magaw, Megan, Keagle, Pamela J., McDonough, Justin A., Boopathy, Sivakumar, Sena-Esteves, Miguel, Nickerson, Jeffrey A., Lutz, Cathleen, Skarnes, William C., Lim, Elaine T., Schafer, Dorothy P., Massi, Francesca, Landers, John E., Bosco, Daryl A.

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EZH2 inhibition reactivates epigenetically silenced FMR1 and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons von Fang, Minggang, Deibler, Sara K, Krishnamurthy, Pranathi Meda, Wang, Feng, Rodriguez, Paola, Banday, Shahid, Virbasius, Ching-Man, Sena-Esteves, Miguel, Watts, Jonathan K, Green, Michael R

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Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis von Latour, Yvonne L., Yoon, Robin, Thomas, Sarah E., Grant, Christina, Li, Cuiling, Sena-Esteves, Miguel, Allende, Maria L., Proia, Richard L., Tifft, Cynthia J.

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Abnormal epiphyseal development in a feline model of Sandhoff disease von McNulty, Margaret A., Prevatt, Patricia B., Nussbaum, Elizabeth R., Randle, Ashley N., Johnson, Aime K., Hudson, Judith A., Gray‐Edwards, Heather L., Sena‐Esteves, Miguel, Martin, Douglas R., Carlson, Cathy S.

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