Disease stages and therapeutic hypotheses in two decades of neurodegenerative disease clinical trials von Mortberg, Meredith A., Vallabh, Sonia M., Minikel, Eric Vallabh

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Analysis of non-human primate models for evaluating prion disease therapeutic efficacy von Mortberg, Meredith A, Minikel, Eric Vallabh, Vallabh, Sonia M

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Towards a treatment for genetic prion disease: trials and biomarkers von Vallabh, Sonia M, Minikel, Eric Vallabh, Schreiber, Stuart L, Lander, Eric S

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Where have prions been all our lives? von Minikel, Eric Vallabh, Vallabh, Sonia M

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The Patient-Scientist’s Mandate von Vallabh, Sonia M

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A single-cell map of antisense oligonucleotide activity in the brain von Mortberg, Meredith A, Gentile, Juliana E, Nadaf, Naeem M, Vanderburg, Charles, Simmons, Sean, Dubinsky, Dan, Slamin, Adam, Maldonado, Salome, Petersen, Caroline L, Jones, Nichole, Kordasiewicz, Holly B, Zhao, Hien T, Vallabh, Sonia M, Minikel, Eric Vallabh

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Prion protein lowering is a disease-modifying therapy across prion disease stages, strains and endpoints von Minikel, Eric Vallabh, Zhao, Hien T, Le, Jason, O’Moore, Jill, Pitstick, Rose, Graffam, Samantha, Carlson, George A, Kavanaugh, Michael P, Kriz, Jasna, Kim, Jae Beom, Ma, Jiyan, Wille, Holger, Aiken, Judd, McKenzie, Deborah, Doh-ura, Katsumi, Beck, Matthew, O’Keefe, Rhonda, Stathopoulos, Jacquelyn, Caron, Tyler, Schreiber, Stuart L, Carroll, Jeffrey B, Kordasiewicz, Holly B, Cabin, Deborah E, Vallabh, Sonia M

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Genetic counseling for prion disease: Updates and best practices von Goldman, Jill S., Vallabh, Sonia M.

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Implications of new genetic risk factors in prion disease von Vallabh, Sonia M., Minikel, Eric Vallabh

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Age at onset in genetic prion disease and the design of preventive clinical trials von Minikel, Eric Vallabh, Vallabh, Sonia M, Orseth, Margaret C, Brandel, Jean-Philippe, Haïk, Stéphane, Laplanche, Jean-Louis, Zerr, Inga, Parchi, Piero, Capellari, Sabina, Safar, Jiri, Kenny, Janna, Fong, Jamie C, Takada, Leonel T, Ponto, Claudia, Hermann, Peter, Knipper, Tobias, Stehmann, Christiane, Kitamoto, Tetsuyuki, Ae, Ryusuke, Hamaguchi, Tsuyoshi, Sanjo, Nobuo, Tsukamoto, Tadashi, Mizusawa, Hidehiro, Collins, Steven J, Chiesa, Roberto, Roiter, Ignazio, de Pedro-Cuesta, Jesús, Calero, Miguel, Geschwind, Michael D, Yamada, Masahito, Nakamura, Yosikazu, Mead, Simon

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Prion protein quantification in human cerebrospinal fluid as a tool for prion disease drug development von Vallabh, Sonia M., Nobuhara, Chloe K., Llorens, Franc, Zerr, Inga, Parchi, Piero, Capellari, Sabina, Kuhn, Eric, Klickstein, Jacob, Safar, Jiri G., Nery, Flavia C., Swoboda, Kathryn J., Geschwind, Michael D., Zetterberg, Henrik, Arnold, Steven E., Minikel, Eric Vallabh, Schreiber, Stuart L.

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Cerebrospinal fluid and plasma biomarkers in individuals at risk for genetic prion disease von Vallabh, Sonia M, Minikel, Eric Vallabh, Williams, Victoria J, Carlyle, Becky C, McManus, Alison J, Wennick, Chase D, Bolling, Anna, Trombetta, Bianca A, Urick, David, Nobuhara, Chloe K, Gerber, Jessica, Duddy, Holly, Lachmann, Ingolf, Stehmann, Christiane, Collins, Steven J, Blennow, Kaj, Zetterberg, Henrik, Arnold, Steven E

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Modulation of prion protein expression through cryptic splice site manipulation von Gentile, Juliana E., Corridon, Taylor L., Mortberg, Meredith A., D’Souza, Elston Neil, Whiffin, Nicola, Minikel, Eric Vallabh, Vallabh, Sonia M.

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Brainwide silencing of prion protein by AAV-mediated delivery of an engineered compact epigenetic editor von Neumann, Edwin N, Bertozzi, Tessa M, Wu, Elaine, Serack, Fiona, Harvey, John W, Brauer, Pamela P, Pirtle, Catherine P, Coffey, Alissa, Howard, Michael, Kamath, Nikita, Lenz, Kenney, Guzman, Kenia, Raymond, Michael H, Khalil, Ahmad S, Deverman, Benjamin E, Minikel, Eric Vallabh, Vallabh, Sonia M, Weissman, Jonathan S

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Domain-specific Quantification of Prion Protein in Cerebrospinal Fluid by Targeted Mass Spectrometry von Minikel, Eric Vallabh, Kuhn, Eric, Cocco, Alexandra R., Vallabh, Sonia M., Hartigan, Christina R., Reidenbach, Andrew G., Safar, Jiri G., Raymond, Gregory J., McCarthy, Michael D., O'Keefe, Rhonda, Llorens, Franc, Zerr, Inga, Capellari, Sabina, Parchi, Piero, Schreiber, Stuart L., Carr, Steven A.

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Therapeutic Trial of anle138b in Mouse Models of Genetic Prion Disease von Vallabh, Sonia M, Zou, Dan, Pitstick, Rose, O'Moore, Jill, Peters, Janet, Silvius, Derek, Kriz, Jasna, Jackson, Walker S, Carlson, George A, Minikel, Eric Vallabh, Cabin, Deborah E

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Fluid Biomarkers in Individuals at Risk for Genetic Prion Disease up to Disease Conversion von Vallabh, Sonia M, Mortberg, Meredith A, Allen, Shona W, Kupferschmid, Ashley C, Kivisakk, Pia, Hammerschlag, Bruno L, Bolling, Anna, Trombetta, Bianca A, Devitte-McKee, Kelli, Ford, Abaigeal M, Sather, Lauren E, Duffy, Griffin, Rivera, Ashley, Gerber, Jessica, McManus, Alison J, Minikel, Eric V, Arnold, Steven E

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Novel quaternary structures of the human prion protein globular domain von Bortot, Leandro Oliveira, Rangel, Victor Lopes, Pavlovici, Francesca A., El Omari, Kamel, Wagner, Armin, Brandao-Neto, Jose, Talon, Romain, von Delft, Frank, Reidenbach, Andrew G., Vallabh, Sonia M., Minikel, Eric Vallabh, Schreiber, Stuart, Nonato, Maria Cristina

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Multimodal small-molecule screening for human prion protein binders von Reidenbach, Andrew G., Mesleh, Michael F., Casalena, Dominick, Vallabh, Sonia M., Dahlin, Jayme L., Leed, Alison J., Chan, Alix I., Usanov, Dmitry L., Yehl, Jenna B., Lemke, Christopher T., Campbell, Arthur J., Shah, Rishi N., Shrestha, Om K., Sacher, Joshua R., Rangel, Victor L., Moroco, Jamie A., Sathappa, Murugappan, Nonato, Maria Cristina, Nguyen, Kong T., Wright, S. Kirk, Liu, David R., Wagner, Florence F., Kaushik, Virendar K., Auld, Douglas S., Schreiber, Stuart L., Minikel, Eric Vallabh

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Antisense oligonucleotides extend survival of prion-infected mice von Raymond, Gregory J, Zhao, Hien Tran, Race, Brent, Raymond, Lynne D, Williams, Katie, Swayze, Eric E, Graffam, Samantha, Le, Jason, Caron, Tyler, Stathopoulos, Jacquelyn, O'Keefe, Rhonda, Lubke, Lori L, Reidenbach, Andrew G, Kraus, Allison, Schreiber, Stuart L, Mazur, Curt, Cabin, Deborah E, Carroll, Jeffrey B, Minikel, Eric Vallabh, Kordasiewicz, Holly, Caughey, Byron, Vallabh, Sonia M

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