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Biorxiv : the Preprint Server for Biology|December 19, 2025
Gene replacement therapy for Piga GPI-anchor deficiency in the developing nervous systemJennifer L Watts, Shibi Likhite, Yoshiko Murakami, et al.
Orphanet Journal of Rare Diseases|November 11, 2022
Sex-split analysis of pathology and motor-behavioral outcomes in a mouse model of CLN8-Batten disease reveals an increased disease burden and trajectory in female Cln8mnd miceAndrew D Holmes, Katherine A White, Melissa A Pratt, et al.
Molecular Therapy. Advances|May 15, 2026
Optimized AAV vector enables potent therapeutic rescue of inherited glycosylphosphatidylinositol deficiency in miceSaori Umeshita, Kae Imanishi, Shibi Likhite, et al.
Biology|June 28, 2023
In Vitro Modeling as a Tool for Testing Therapeutics for Spinal Muscular Atrophy and IGHMBP2-Related DisordersJulieth Andrea Sierra-Delgado, Shrestha Sinha-Ray, Abuzar Kaleem, et al.
Molecular Therapy. Methods & Clinical Development|January 16, 2024
AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse modelsYoshiko Murakami, Saori Umeshita, Kae Imanishi, et al.
Molecular Therapy. Methods & Clinical Development|July 18, 2024
The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndromeShin Jeon, Jaein Park, Shibi Likhite, et al.
Molecular Therapy. Methods & Clinical Development|September 25, 2023
In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humansMaura K Schwartz, Shibi Likhite, Tatyana A Vetter, et al.
Cells|April 12, 2022
Neurotoxic Astrocytes Directly Converted from Sporadic and Familial ALS Patient Fibroblasts Reveal Signature Diversities and miR-146a Theragnostic Potential in Specific SubtypesCátia Gomes, Catarina Sequeira, Shibi Likhite, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 7, 2013
Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALSKevin D Foust, Desirée L Salazar, Shibi Likhite, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience|November 21, 2014
Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortexGretchen M Thomsen, Genevieve Gowing, Jessica Latter, et al.
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