Science & Technology
Publications
<u>Publications</u>

Publications

Martin L. Brady, Raghu Raghavan, Deep Singh, P.J. Anand, Adam Fleisher, Jaime Mata, William C. Broaddus, William L. Olbricht In vivo performance of a microfabricated catheter for intraparenchymal delivery Journal of Neuroscience Methods, 2014.
Khani M, Burla GKR, Sass LR, Arters ON, Xing T, Wu H, Martin BA. Human in silico trials for parametric computational fluid dynamics investigation of cerebrospinal fluid drug delivery: impact of injection location, injection protocol, and physiology. Fluids and Barriers of the CNS, 2022.
Gosse M, Jones C, Jesus D, D’Costa S. Regulatory & supply chain implications for plasmids as critical starting materials in the manufacture of viral vector gene therapy products. Cell & Gene Therapy Insights, 2022.
Przanowski P, Wasko U, Zheng Z, Yu J, Sherman R, Zhu LJ, McConnell MJ, Tushir-Singh J, Green MR, Bhatnagar S. Pharmacological reactivation of inactive X-linked Mecp2 in cerebral cortical neurons of living mice. Proceedings of the National Academy of Sciences of the USA, 2018.
Seiner A, Burla GKR, Shrestha D, Bowen M, Horvath JD, Martin BA. Investigation of Human Intrathecal Solute Transport Dynamics Using a Novel in vitro Cerebrospinal Fluid System Analog. Frontiers in Neuroimaging, 2022.
Sass LR, Khani M, Romm J, Schmid Daners M, McCain K, Freeman T, Carter GT, Weeks DL, Petersen B, Aldred J, Wingett D, Martin BA. Non-invasive MRI quantification of cerebrospinal fluid dynamics in amyotrophic lateral sclerosis patients. Fluids and Barriers of the CNS, 2020.
Khani M, Sass LR, Sharp MK, McCabe AR, Zitella Verbick LM, Lad SP, Martin BA. In vitro and numerical simulation of blood removal from cerebrospinal fluid: comparison of lumbar drain to Neurapheresis therapy. Fluids and Barriers of the CNS, 2020.
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Single-dose gene-replacement therapy for Spinal Muscular Atrophy The New England Journal of Medicine, 2017.

Posters & Presentations

Sierra-Delgado JA, Likhite S, McGovern V, Huffenberger A, Hicks AN, Holbrook SE, Nizzardo M, Dennys-Rivers C, Ray S, Corti S, Burghes A, Arnold WD, Cox G, Meyer K. Evaluation of AAV9 gene therapy for SMARD1/CMT2S in different mouse models reveal differences in efficacy dependent on promoter choice. Presented at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, 2022.
Gosse M, Likhite S, Jesus D, Meyer K, D’Costa S. Development of an upstream process and analytics for AAV manufacturing. Presented at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, 2022.
Sierra-Delgado JA, Likhite S, McGovern V, Huffenberger A, Hicks AN, Holbrook SE, Nizzardo M, Dennys-Rivers C, Ray S, Corti S, Burghes A, Arnold WD, Cox G, Meyer K. Multicenter AAV gene therapy studies for SMARD1/CMT2S establish safety and efficacy in multiple animal models and pave the way for initiation of a Phase I/II clinical trial. Presented at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, 2022.
Powers S*, Lou S, Nardini S, Likhite S, Sierra-Delgado JA, Baird M, Roussel F, Kaleem A, Schwartz M, Ray S, Bhatnagar S, Meyer K. A novel gene therapy for Rett Syndrome through reactivation of the silent X chromosome. Presented at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, 2022. *Received Excellence in Research Award