Additive amelioration of ALS by co‐targeting independent pathogenic mechanisms

por Frakes, Ashley E., Braun, Lyndsey, Ferraiuolo, Laura, Guttridge, Denis C., Kaspar, Brian K.
Publicado 2017

IGF-I instructs multipotent adult neural progenitor cells to become oligodendrocytes

por Hsieh, Jenny, Aimone, James B., Kaspar, Brian K., Kuwabara, Tomoko, Nakashima, Kinichi, Gage, Fred H.
Publicado 2004

Two Factor Reprogramming of Human Neural Stem Cells into Pluripotency

por Hester, Mark E., Song, SungWon, Miranda, Carlos J., Eagle, Amy, Schwartz, Phillip H., Kaspar, Brian K.
Publicado 2009

NEUROD1 Instructs Neuronal Conversion in Non-Reactive Astrocytes

por Brulet, Rebecca, Matsuda, Taito, Zhang, Ling, Miranda, Carlos, Giacca, Mauro, Kaspar, Brian K., Nakashima, Kinichi, Hsieh, Jenny
Publicado 2017

AAV1.NT-3 Gene Therapy for Charcot–Marie–Tooth Neuropathy

por Sahenk, Zarife, Galloway, Gloria, Clark, Kelly Reed, Malik, Vinod, Rodino-Klapac, Louise R, Kaspar, Brian K., Chen, Lei, Braganza, Cilwyn, Montgomery, Chrystal, Mendell, Jerry R
Publicado 2014

Neurotoxic Astrocytes Directly Converted from Sporadic and Familial ALS Patient Fibroblasts Reveal Signature Diversities and miR-146a Theragnostic Potential in Specific Subtypes

por Gomes, Cátia, Sequeira, Catarina, Likhite, Shibi, Dennys, Cassandra N., Kolb, Stephen J., Shaw, Pamela J., Vaz, Ana R., Kaspar, Brian K., Meyer, Kathrin, Brites, Dora
Publicado 2022

Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model

por Nizzardo, Monica, Simone, Chiara, Rizzo, Federica, Salani, Sabrina, Dametti, Sara, Rinchetti, Paola, Del Bo, Roberto, Foust, Kevin, Kaspar, Brian K., Bresolin, Nereo, Comi, Giacomo P., Corti, Stefania
Publicado 2015

Potent spinal parenchymal AAV9-mediated gene delivery by subpial injection in adult rats and pigs

por Miyanohara, Atsushi, Kamizato, Kota, Juhas, Stefan, Juhasova, Jana, Navarro, Michael, Marsala, Silvia, Lukacova, Nada, Hruska-Plochan, Marian, Curtis, Erik, Gabel, Brandon, Ciacci, Joseph, Ahrens, Eric T, Kaspar, Brian K, Cleveland, Don, Marsala, Martin
Publicado 2016

Electrophysiological biomarkers in spinal muscular atrophy: proof of concept

por David Arnold, W, Porensky, Paul N, McGovern, Vicki L, Iyer, Chitra C, Duque, Sandra, Li, Xiaobai, Meyer, Kathrin, Schmelzer, Leah, Kaspar, Brian K, Kolb, Stephen J, Kissel, John T, Burghes, Arthur H M
Publicado 2014

MHC class I protects motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis (ALS)

por Song, SungWon, Miranda, Carlos J., Braun, Lyndsey, Meyer, Kathrin, Frakes, Ashley E., Ferraiuolo, Laura, Likhite, Shibi, Bevan, Adam K., Foust, Kevin D., McConnell, Michael J., Walker, Christopher M., Kaspar, Brian K.
Publicado 2016

Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease

por Johnson, Tyler B., Brudvig, Jon J., Likhite, Shibi, Pratt, Melissa A., White, Katherine A., Cain, Jacob T., Booth, Clarissa D., Timm, Derek J., Davis, Samantha S., Meyerink, Brandon, Pineda, Ricardo, Dennys-Rivers, Cassandra, Kaspar, Brian K., Meyer, Kathrin, Weimer, Jill M.
Publicado 2023

Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS

por Iannitti, Tommaso, Scarrott, Joseph M., Likhite, Shibi, Coldicott, Ian R.P., Lewis, Katherine E., Heath, Paul R., Higginbottom, Adrian, Myszczynska, Monika A., Milo, Marta, Hautbergue, Guillaume M., Meyer, Kathrin, Kaspar, Brian K., Ferraiuolo, Laura, Shaw, Pamela J., Azzouz, Mimoun
Publicado 2018

Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice

por White, Katherine A., Nelvagal, Hemanth R., Poole, Timothy A., Lu, Bin, Johnson, Tyler B., Davis, Samantha, Pratt, Melissa A., Brudvig, Jon, Assis, Ana B., Likhite, Shibi, Meyer, Kathrin, Kaspar, Brian K., Cooper, Jonathan D., Wang, Shaomei, Weimer, Jill M.
Publicado 2021

The C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy

por Webster, Christopher P, Smith, Emma F, Bauer, Claudia S, Moller, Annekathrin, Hautbergue, Guillaume M, Ferraiuolo, Laura, Myszczynska, Monika A, Higginbottom, Adrian, Walsh, Matthew J, Whitworth, Alexander J, Kaspar, Brian K, Meyer, Kathrin, Shaw, Pamela J, Grierson, Andrew J, De Vos, Kurt J
Publicado 2016

Systemic Gene Delivery Transduces the Enteric Nervous System of Guinea Pigs and Cynomolgus Macaques

por Gombash, Sara E, Cowley, Christopher J, Fitzgerald, Julie A, Lepak, Christina A, Neides, Mitchell G, Hook, Kathryn, Todd, Levi J, Wang, Guo-Du, Mueller, Christian, Kaspar, Brian K, Bielefeld, Eric C, Fischer, Andrew J, Wood, Jackie D, Foust, Kevin D
Publicado 2017

Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

por Bevan, Adam K, Duque, Sandra, Foust, Kevin D, Morales, Pablo R, Braun, Lyndsey, Schmelzer, Leah, Chan, Curtis M, McCrate, Mary, Chicoine, Louis G, Coley, Brian D, Porensky, Paul N, Kolb, Stephen J, Mendell, Jerry R, Burghes, Arthur HM, Kaspar, Brian K
Publicado 2011

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease

por Cain, Jacob T., Likhite, Shibi, White, Katherine A., Timm, Derek J., Davis, Samantha S., Johnson, Tyler B., Dennys-Rivers, Cassandra N., Rinaldi, Federica, Motti, Dario, Corcoran, Sarah, Morales, Pablo, Pierson, Christopher, Hughes, Stephanie M., Lee, Stella Y., Kaspar, Brian K., Meyer, Kathrin, Weimer, Jill M.
Publicado 2019

Astrocytes from Familial and Sporadic ALS Patients are Toxic to Motor Neurons

por Haidet-Phillips, Amanda M., Hester, Mark E., Miranda, Carlos J., Meyer, Kathrin, Braun, Lyndsey, Frakes, Ashley, Song, SungWon, Likhite, Shibi, Murtha, Matthew J., Foust, Kevin D., Rao, Meghan, Eagle, Amy, Kammesheidt, Anja, Christensen, Ashley, Mendell, Jerry R., Burghes, Arthur H.M., Kaspar, Brian K.
Publicado 2011

Gene delivery to the spinal cord using MRI-guided focused ultrasound

por Weber-Adrian, Danielle, Thévenot, Emmanuel, O'Reilly, Meaghan A., Oakden, Wendy, Akens, Margarete K., Ellens, Nicholas, Markham-Coultes, Kelly, Burgess, Alison, Finkelstein, Joel, Yee, Albert J.M., Whyne, Cari M., Foust, Kevin D., Kaspar, Brian K., Stanisz, Greg J., Chopra, Rajiv, Hynynen, Kullervo, Aubert, Isabelle
Publicado 2015

SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits

por Hautbergue, Guillaume M., Castelli, Lydia M., Ferraiuolo, Laura, Sanchez-Martinez, Alvaro, Cooper-Knock, Johnathan, Higginbottom, Adrian, Lin, Ya-Hui, Bauer, Claudia S., Dodd, Jennifer E., Myszczynska, Monika A., Alam, Sarah M., Garneret, Pierre, Chandran, Jayanth S., Karyka, Evangelia, Stopford, Matthew J., Smith, Emma F., Kirby, Janine, Meyer, Kathrin, Kaspar, Brian K., Isaacs, Adrian M., El-Khamisy, Sherif F., De Vos, Kurt J., Ning, Ke, Azzouz, Mimoun, Whitworth, Alexander J., Shaw, Pamela J.
Publicado 2017