Publications by authors named "Shibi Likhite"

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Gene replacement therapy for Piga GPI-anchor deficiency in the developing nervous system.
Jennifer L Watts, Shibi Likhite, Yoshiko Murakami, Taroh Kinoshita, Kathrin Meyer

bioRxiv· December 2025

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Targeting microRNA-dependent control of X chromosome inactivation improves the Rett Syndrome phenotype.
Song Lou, Rachisan DJiake Tihagam, Urszula N Wasko, Zaffar Equbal, Sanjay Venkatesan, Shibi Likhite

Nat Commun· July 2025

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The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome.
Shin Jeon, Jaein Park, Shibi Likhite, Ji Hwan Moon, Dongjun Shin

Mol Ther Methods Clin Dev· September 2024

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Combination AAV therapy with galectin-1 and SOD1 downregulation demonstrates superior therapeutic effect in a severe ALS mouse model.
Megan C Baird, Shibi B Likhite, Tatyana A Vetter, Joseph R Caporale, Holly B Girard

Mol Ther Methods Clin Dev· September 2024

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AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models.
Yoshiko Murakami, Saori Umeshita, Kae Imanishi, Yoshichika Yoshioka, Akinori Ninomiya, Shibi Likhite

Mol Ther Methods Clin Dev· March 2024

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Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates.
Samantha Powers, Shibi Likhite, Kamal K Gadalla, Carlos J Miranda, Amy J Huffenberger

Mol Ther· September 2023

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In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans.
Maura K Schwartz, Shibi Likhite, Tatyana A Vetter, Megan C Baird, Vicki McGovern

Mol Ther Methods Clin Dev· September 2023

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In Vitro Modeling as a Tool for Testing Therapeutics for Spinal Muscular Atrophy and IGHMBP2-Related Disorders.
Julieth Andrea Sierra-Delgado, Shrestha Sinha-Ray, Abuzar Kaleem, Meysam Ganjibakhsh, Mohini Parvate, Shibi Likhite

Biology (Basel)· June 2023

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Prevalence of Neutralizing Antibodies against Adeno-Associated Virus Serotypes 1, 2, and 9 in Non-Injected Latin American Patients with Heart Failure-ANVIAS Study.
Julieth A Sierra-Delgado, Shibi Likhite, Paula K Bautista, Sergio A Gómez-Ochoa, Luis E Echeverría

Int J Mol Sci· March 2023

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Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease.
Tyler B Johnson, Jon J Brudvig, Shibi Likhite, Melissa A Pratt, Katherine A White

Front Genet· March 2023

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Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy.
Shrestha Sinha Ray, Debdeep Dutta, Cassandra Dennys, Samantha Powers, Florence Roussel, Shibi Likhite

Cell Rep· December 2022

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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 Cln8 mice.
Andrew D Holmes, Katherine A White, Melissa A Pratt, Tyler B Johnson, Shibi Likhite

Orphanet J Rare Dis· November 2022

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AAV9-MCT8 Delivery at Juvenile Stage Ameliorates Neurological and Behavioral Deficits in a Mouse Model of MCT8-Deficiency.
Xiao-Hui Liao, Pablo Avalos, Oksana Shelest, Raz Ofan, Michael Shilo, Shibi Likhite

Thyroid· July 2022

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Optimization and validation of CAR transduction into human primary NK cells using CRISPR and AAV.
Meisam Naeimi Kararoudi, Shibi Likhite, Ezgi Elmas, Kenta Yamamoto, Maura Schwartz

Cell Rep Methods· June 2022

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Neurotoxic Astrocytes Directly Converted from Sporadic and Familial ALS Patient Fibroblasts Reveal Signature Diversities and miR-146a Theragnostic Potential in Specific Subtypes.
Cátia Gomes, Catarina Sequeira, Shibi Likhite, Cassandra N Dennys, Stephen J Kolb

Cells· April 2022

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Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.
Katherine A White, Hemanth R Nelvagal, Timothy A Poole, Bin Lu, Tyler B Johnson, Shibi Likhite

Mol Ther Methods Clin Dev· March 2021

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AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease.
Tyler B Johnson, Katherine A White, Jon J Brudvig, Jacob T Cain, Logan Langin, Shibi Likhite

Mol Ther· January 2021

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Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.
Jacob T Cain, Shibi Likhite, Katherine A White, Derek J Timm, Samantha S Davis

Mol Ther· October 2019

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Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS.
Tommaso Iannitti, Joseph M Scarrott, Shibi Likhite, Ian R P Coldicott, Katherine E Lewis

Mol Ther Nucleic Acids· September 2018

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Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism.
Laura Ferraiuolo, Kathrin Meyer, Thomas W Sherwood, Jonathan Vick, Shibi Likhite

Proc Natl Acad Sci U S A· October 2016

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Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis.
SungWon Song, Carlos J Miranda, Lyndsey Braun, Kathrin Meyer, Ashley E Frakes, Shibi Likhite

Nat Med· April 2016

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Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
Kathrin Meyer, Laura Ferraiuolo, Leah Schmelzer, Lyndsey Braun, Vicki McGovern, Shibi Likhite

Mol Ther· March 2015

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Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex.
Gretchen M Thomsen, Genevieve Gowing, Jessica Latter, Maximus Chen, Jean-Philippe Vit, Shibi Likhite

J Neurosci· November 2014

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Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS.
Kathrin Meyer, Laura Ferraiuolo, Carlos J Miranda, Shibi Likhite, Sohyun McElroy

Proc Natl Acad Sci U S A· January 2014

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