Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
Meyer, Kathrin, Ferraiuolo, Laura, Schmelzer, Leah, Braun, Lyndsey, McGovern, Vicki, Likhite, Shibi, Michels, Olivia, Govoni, Alessandra, Fitzgerald, Julie, Morales, Pablo, Foust, Kevin D, Mendell, Jerry R, Burghes, Arthur H M, Kaspar, Brian K
Published in Molecular therapy (01.03.2015)
Published in Molecular therapy (01.03.2015)
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Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism
Ferraiuolo, Laura, Meyer, Kathrin, Sherwood, Thomas W., Vick, Jonathan, Likhite, Shibi, Frakes, Ashley, Miranda, Carlos J., Braun, Lyndsey, Heath, Paul R., Pineda, Ricardo, Beattie, Christine E., Shaw, Pamela J., Askwith, Candice C., McTigue, Dana, Kaspar, Brian K.
Published in Proceedings of the National Academy of Sciences - PNAS (18.10.2016)
Published in Proceedings of the National Academy of Sciences - PNAS (18.10.2016)
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Astrocytes from familial and sporadic ALS patients are toxic to motor neurons
Kaspar, Brian K, 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
Published in Nature biotechnology (10.08.2011)
Published in Nature biotechnology (10.08.2011)
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Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS
Foust, Kevin D, Salazar, Desirée L, Likhite, Shibi, Ferraiuolo, Laura, Ditsworth, Dara, Ilieva, Hristelina, Meyer, Kathrin, Schmelzer, Leah, Braun, Lyndsey, Cleveland, Don W, Kaspar, Brian K
Published in Molecular therapy (01.12.2013)
Published in Molecular therapy (01.12.2013)
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Journal Article
Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis
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
Published in Nature medicine (01.04.2016)
Published in Nature medicine (01.04.2016)
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A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy
Mendell, Jerry R, Sahenk, Zarife, Malik, Vinod, Gomez, Ana M, Flanigan, Kevin M, Lowes, Linda P, Alfano, Lindsay N, Berry, Katherine, Meadows, Eric, Lewis, Sarah, Braun, Lyndsey, Shontz, Kim, Rouhana, Maria, Clark, Kelly Reed, Rosales, Xiomara Q, Al-Zaidy, Samiah, Govoni, Alessandra, Rodino-Klapac, Louise R, Hogan, Mark J, Kaspar, Brian K
Published in Molecular therapy (01.01.2015)
Published in Molecular therapy (01.01.2015)
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Aging brain microenvironment decreases hippocampal neurogenesis through Wnt‐mediated survivin signaling
Miranda, Carlos J., Braun, Lyndsey, Jiang, Yuying, Hester, Mark E., Zhang, Ling, Riolo, Matthew, Wang, Haijuan, Rao, Meghan, Altura, Rachel A., Kaspar, Brian K.
Published in Aging cell (01.06.2012)
Published in Aging cell (01.06.2012)
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Journal Article
Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery
Bevan, Adam K., Hutchinson, Kirk R., Foust, Kevin D., Braun, Lyndsey, McGovern, Vicki L., Schmelzer, Leah, Ward, Jennifer G., Petruska, Jeffrey C., Lucchesi, Pamela A., Burghes, Arthur H.M., Kaspar, Brian K.
Published in Human molecular genetics (16.07.2010)
Published in Human molecular genetics (16.07.2010)
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Adeno Associated Virus 9-Based Gene Therapy Delivers a Functional Monocarboxylate Transporter 8, Improving Thyroid Hormone Availability to the Brain of Mct8-Deficient Mice
Iwayama, Hideyuki, Liao, Xiao-Hui, Braun, Lyndsey, Bárez-López, Soledad, Kaspar, Brian, Weiss, Roy E, Dumitrescu, Alexandra M, Guadaño-Ferraz, Ana, Refetoff, Samuel
Published in Thyroid (New York, N.Y.) (01.09.2016)
Published in Thyroid (New York, N.Y.) (01.09.2016)
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Journal Article
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, Jerry R., Al-Zaidy, Samiah, Shell, Richard, Arnold, W. Dave, Rodino-Klapac, Louise R., Prior, Thomas W., Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T., Nagendran, Sukumar, L’Italien, James, Sproule, Douglas M., Wells, Courtney, Cardenas, Jessica A., Heitzer, Marjet D., Kaspar, Allan, Corcoran, Sarah, Braun, Lyndsey, Likhite, Shibi, Miranda, Carlos, Meyer, Kathrin, Foust, K.D., Burghes, Arthur H.M., Kaspar, Brian K.
Published in The New England journal of medicine (02.11.2017)
Published in The New England journal of medicine (02.11.2017)
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Journal Article
RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
Foust, Kevin D, Wang, Xueyong, McGovern, Vicki L, Braun, Lyndsey, Bevan, Adam K, Haidet, Amanda M, Le, Thanh T, Morales, Pablo R, Rich, Mark M, Burghes, Arthur H M, Kaspar, Brian K
Published in Nature biotechnology (28.02.2010)
Published in Nature biotechnology (28.02.2010)
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Journal Article
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, Jerry R, Al-Zaidy, Samiah, Shell, Richard, Arnold, W. Dave, Rodino-Klapac, Louise R, Prior, Thomas W, Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T, Nagendran, Sukumar, L’Italien, James, Sproule, Douglas M, Wells, Courtney, Cardenas, Jessica A, Heitzer, Marjet D, Kaspar, Allan, Corcoran, Sarah, Braun, Lyndsey, Likhite, Shibi, Miranda, Carlos, Meyer, Kathrin, Foust, K.D, Burghes, Arthur H.M, Kaspar, Brian K
Published in The New England journal of medicine (02.11.2017)
Published in The New England journal of medicine (02.11.2017)
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Journal Article
Microglia Induce Motor Neuron Death via the Classical NF-κB Pathway in Amyotrophic Lateral Sclerosis
Frakes, Ashley E., Ferraiuolo, Laura, Haidet-Phillips, Amanda M., Schmelzer, Leah, Braun, Lyndsey, Miranda, Carlos J., Ladner, Katherine J., Bevan, Adam K., Foust, Kevin D., Godbout, Jonathan P., Popovich, Phillip G., Guttridge, Denis C., Kaspar, Brian K.
Published in Neuron (Cambridge, Mass.) (05.03.2014)
Published in Neuron (Cambridge, Mass.) (05.03.2014)
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Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
Kaspar, Brian K, Foust, Kevin D, Wang, Xueyong, McGovern, Vicki L, Braun, Lyndsey, Bevan, Adam K, Haidet, Amanda M, Le, Thanh T, Morales, Pablo R, Rich, Mark M, Burghes, Arthur H M
Published in Nature biotechnology (01.03.2010)
Published in Nature biotechnology (01.03.2010)
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Journal Article
Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders
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
Published in Molecular therapy (01.11.2011)
Published in Molecular therapy (01.11.2011)
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Retraction Note: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
Foust, Kevin D., Wang, Xueyong, McGovern, Vicki L., Braun, Lyndsey, Bevan, Adam K., Haidet, Amanda M., Le, Thanh T., Morales, Pablo R., Rich, Mark M., Burghes, Arthur H. M., Kaspar, Brian K.
Published in Nature biotechnology (01.11.2022)
Published in Nature biotechnology (01.11.2022)
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Journal Article
MHC class I protects motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis (ALS)
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.
Published in Nature medicine (29.02.2016)
Published in Nature medicine (29.02.2016)
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Journal Article
480. Gene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional Outcomes
Mendell, Jerry R., Al-Zaidy, Samiah, Shell, Richard, Arnold, W Dave, Rodino-Klapac, Louise, Kissel, John T., Prior, Thomas W., Miranda, Carlos, Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Petek, Christopher, Church, Kathleen, Braun, Lyndsey, Corcoran, Sarah, Meyer, Kathrin, Likhite, Shibi, Burghes, Arthur HM, Foust, Kevin D., Kaspar, Brian K.
Published in Molecular therapy (01.05.2016)
Published in Molecular therapy (01.05.2016)
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Microglia Induce Motor Neuron Death via the Classical NF-[kappa]B Pathway in Amyotrophic Lateral Sclerosis
Frakes, Ashley E, Ferraiuolo, Laura, Haidet-Phillips, Amanda M, Schmelzer, Leah, Braun, Lyndsey, Miranda, Carlos J, Ladner, Katherine J, Bevan, Adam K, Foust, Kevin D, Godbout, Jonathan P, Popovich, Phillip G, Guttridge, Denis C, Kaspar, Brian K
Published in Neuron (Cambridge, Mass.) (05.03.2014)
Published in Neuron (Cambridge, Mass.) (05.03.2014)
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