Research:
My work is focused on the implementation of a novel gene therapy treatment for Rett syndrome. We have developed an Adeno-associated virus serotype 9 (AAV9) vector to deliver human MeCP2 cNDA under the control of an endogenous promoter. We are currently testing the safety and efficacy of treatment with vector in a mouse model of the disease as well as healthy non-human primates. The goal of this work is to initiate a clinical trial of our vector for Rett syndrome patients in the near future.
Awards:
2017 - RITA Graduate Fellowship, Nationwide Chidlren’s
Publications:
Powers, S., Kwok, S., Lovejoy, E., Lavin, T., Sher, R., (2017) “Embryonic Exposure to the Environmental Neurotoxin BMAA Negatively Impacts Early Neuronal Development and Progression of Neurodegeneration in the SOD1-G93R Zebrafish Model of Amyotrophic Lateral Sclerosis.” Toxicol Sci (2017) 157 (1): 129-140
Presentations:
Samantha Powers, Carlos J, Miranda, Cassandra Dennys, Amy Huffenberger, Stephanie Solano, Katelynn Kinley, Kimberly Sandy, Lyndsey Braun, Kathrin Meyer, Kevin Foust, Brian K. Kaspar (2017 May) Gene Therapy for Rett Syndrome Rescues MeCP2 Null Mice Talk Presented at the 2017 Interdisciplinary Graduate Programs Symposium, Columbus Ohio
Samantha Powers, Carlos J, Miranda, Cassandra Dennys, Amy Huffenberger, Stephanie Solano, Katelynn Kinley, Kimberly Sandy, Lyndsey Braun, Kathrin Meyer, Kevin Foust, Brian K. Kaspar (2017 April) Gene Therapy for Rett Syndrome Rescues MeCP2 Null Mice Poster Presented at the April 2017 Wexner Medical Center Trainee Research Day, Columbus Ohio
Samantha Powers, Carlos J, Miranda, Cassandra Dennys, Amy Huffenberger, Stephanie Solano, Katelynn Kinley, Kimberly Sandy, Lyndsey Braun, Kathrin Meyer, Kevin Foust, Brian K. Kaspar (2017 March) Rett Syndrome Gene Therapy Improves Survival and Ameliorates Behavioral Phenotypes in MeCP2 Null Mice Talk Presented at the February 2017 31st Annual Edward F. Hayes Graduate Research Forum, Columbus Ohio
Samantha Powers, Carlos J, Miranda, Cassandra Dennys, Amy Huffenberger, Stephanie Solano, Katelynn Kinley, Kimberly Sandy, Lyndsey Braun, Kathrin Meyer, Kevin Foust, Brian K. Kaspar (2017 February) Gene Therapy for Rett Syndrome Rescues MeCP2 Null Mice Poster Presented at the February 2017 Nationwide Children’s Hospital Poster of the Week, Columbus Ohio
Samantha Powers, Carlos J, Miranda, Cassandra Dennys, Amy Huffenberger, Stephanie Solano, Lyndsey Braun, Kathrin Meyer, Kevin Foust, Brian K. Kaspar (2016 November) Gene Therapy for Rett Syndrome Rescues MeCP2 Null Mice Talk Presented at the 2016 Nationwide Children’s Hospital Research Retreat, Columbus Ohio
Gene Therapy for Rett Syndrome IGP Symposium, May 18, 2016
