Adeno-associated viral vector delivered RNAi for gene therapy of SOD1 Amyotrophic Lateral Sclerosis

by Lorelei Stoica, Lorelei Stoica, Miguel Sena-Esteves, Miguel Sena-Esteves
Published 2016-08-01

Bis(monoacylglycero)phosphate: a secondary storage lipid in the gangliosidoses

by Zeynep Akgoc, Miguel Sena-Esteves, Douglas R. Martin, Xianlin Han, Alessandra d'Azzo, Thomas N. Seyfried
Published 2015-05-01

Functional Coexpression of HSV-1 Thymidine Kinase and Green Fluorescent Protein: Implications for Noninvasive Imaging of Transgene Expression

by Andreas Jacobs, Michael Dubrovin, Jeff Hewett, Miguel Sena-Esteves, Cui-Wen Tan, Mark Slack, Michele Sadelain, Xandra O. Breakefield, Juri G. Tjuvajev
Published 1999-06-01

Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis

by Yvonne L. Latour, Robin Yoon, Sarah E. Thomas, Christina Grant, Cuiling Li, Miguel Sena-Esteves, Maria L. Allende, Richard L. Proia, Cynthia J. Tifft
Published 2019-12-01

AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival.

by Rena C Baek, Marike L D Broekman, Stanley G Leroy, Laryssa A Tierney, Michael A Sandberg, Alessandra d'Azzo, Thomas N Seyfried, Miguel Sena-Esteves
Published 2010-10-01

Triple Bioluminescence Imaging for In Vivo Monitoring of Cellular Processes

by Casey A Maguire, M Sarah Bovenberg, Matheus HW Crommentuijn, Johanna M Niers, Mariam Kerami, Jian Teng, Miguel Sena-Esteves, Christian E Badr, Bakhos A Tannous
Published 2013-01-01

Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation[S]

by Maria L. Allende, Emily K. Cook, Bridget C. Larman, Adrienne Nugent, Jacqueline M. Brady, Diane Golebiowski, Miguel Sena-Esteves, Cynthia J. Tifft, Richard L. Proia
Published 2018-03-01

Survival benefit and phenotypic improvement by hamartin gene therapy in a tuberous sclerosis mouse brain model

by Shilpa Prabhakar, Xuan Zhang, June Goto, Sangyeul Han, Charles Lai, Roderick Bronson, Miguel Sena-Esteves, Vijaya Ramesh, Anat Stemmer-Rachamimov, David J. Kwiatkowski, Xandra O. Breakefield
Published 2015-10-01

Correction: Stochastic Model of Tsc1 Lesions in Mouse Brain

by Shilpa Prabhakar, June Goto, Xuan Zhang, Miguel Sena-Esteves, Roderick Bronson, Jillian Brockmann, Davide Gianni, Gregory R. Wojtkiewicz, John W. Chen, Anat Stemmer-Rachamimov, David J. Kwiatkowski, Xandra O. Breakefield
Published 2013-01-01

Stochastic model of Tsc1 lesions in mouse brain.

by Shilpa Prabhakar, June Goto, Xuan Zhang, Miguel Sena-Esteves, Roderick Bronson, Jillian Brockmann, Davide Gianni, Gregory R Wojtkiewicz, John W Chen, Anat Stemmer-Rachamimov, David J Kwiatkowski, Xandra O Breakefield
Published 2013-01-01

Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy

by Heather L. Gray-Edwards, Xuntian Jiang, Ashley N. Randle, Amanda R. Taylor, Taylor L. Voss, Aime K. Johnson, Victoria J. McCurdy, Miguel Sena-Esteves, Daniel S. Ory, Douglas R. Martin
Published 2017-09-01

Correction: Stochastic Model of Lesions in Mouse Brain.

by Shilpa Prabhakar, June Goto, Xuan Zhang, Miguel Sena-Esteves, Roderick Bronson, Jillian Brockmann, Davide Gianni, Gregory R. Wojtkiewicz, John W. Chen, Anat Stemmer-Rachamimov, David J. Kwiatkowski, Xandra O. Breakefield
Published 2013-01-01

Upregulating β-hexosaminidase activity in rodents prevents α-synuclein lipid associations and protects dopaminergic neurons from α-synuclein-mediated neurotoxicity

by Oeystein R. Brekk, Joanna A. Korecka, Cecile C. Crapart, Mylene Huebecker, Zachary K. MacBain, Sara Ann Rosenthal, Miguel Sena-Esteves, David A. Priestman, Frances M. Platt, Ole Isacson, Penelope J. Hallett
Published 2020-08-01

Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1

by Shilpa Prabhakar, Pike See Cheah, Xuan Zhang, Max Zinter, Maria Gianatasio, Eloise Hudry, Roderick T. Bronson, David J. Kwiatkowski, Anat Stemmer-Rachamimov, Casey A. Maguire, Miguel Sena-Esteves, Bakhos A. Tannous, Xandra O. Breakefield
Published 2019-12-01

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

by Dan Wang, Li Zhong, Mengxin Li, Jia Li, Karen Tran, Lingzhi Ren, Ran He, Jun Xie, Richard P. Moser, Cara Fraser, Tim Kuchel, Miguel Sena-Esteves, Terence R. Flotte, Neil Aronin, Guangping Gao
Published 2018-12-01

Induced Pluripotent Stem Cell Models of Progranulin-Deficient Frontotemporal Dementia Uncover Specific Reversible Neuronal Defects

by Sandra Almeida, Zhijun Zhang, Giovanni Coppola, Wenjie Mao, Kensuke Futai, Anna Karydas, Michael D. Geschwind, M. Carmela Tartaglia, Fuying Gao, Davide Gianni, Miguel Sena-Esteves, Daniel H. Geschwind, Bruce L. Miller, Robert V. Farese, Jr., Fen-Biao Gao
Published 2012-10-01

Induced Pluripotent Stem Cell Models of Progranulin-Deficient Frontotemporal Dementia Uncover Specific Reversible Neuronal Defects

by Sandra Almeida, Zhijun Zhang, Giovanni Coppola, Wenjie Mao, Kensuke Futai, Anna Karydas, Michael D. Geschwind, M. Carmela Tartaglia, Fuying Gao, Davide Gianni, Miguel Sena-Esteves, Daniel H. Geschwind, Bruce L. Miller, Robert V. Farese, Jr., Fen-Biao Gao
Published 2012-11-01

Selection of an Efficient AAV Vector for Robust CNS Transgene Expression

by Killian S. Hanlon, Jonah C. Meltzer, Tetyana Buzhdygan, Ming J. Cheng, Miguel Sena-Esteves, Rachel E. Bennett, Timothy P. Sullivan, Roshanak Razmpour, Yi Gong, Carrie Ng, Josette Nammour, Daniela Maiz, Simon Dujardin, Servio H. Ramirez, Eloise Hudry, Casey A. Maguire
Published 2019-12-01

Switching on the lights for gene therapy.

by Alexandra Winkeler, Miguel Sena-Esteves, Leonie E M Paulis, Hongfeng Li, Yannic Waerzeggers, Benedikt Rückriem, Uwe Himmelreich, Markus Klein, Parisa Monfared, Maria A Rueger, Michael Heneka, Stefan Vollmar, Mathias Hoehn, Cornel Fraefel, Rudolf Graf, Klaus Wienhard, Wolf D Heiss, Andreas H Jacobs
Published 2007-01-01

Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis

by Toloo Taghian, Ana Rita Batista, Sarah Kamper, Michael Caldwell, Laura Lilley, Hao Li, Paola Rodriguez, Katerina Mesa, Shaokuan Zheng, Robert M. King, Matthew J. Gounis, Sophia Todeasa, Anne Maguire, Douglas R. Martin, Miguel Sena-Esteves, Thomas J. Meade, Heather L. Gray-Edwards
Published 2021-12-01