Publications by authors named "Gray Edwards"

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Five-year analysis of efficacy and safety of a bidirectional AAV gene therapy in Tay-Sachs sheep.
Toloo Taghian, Jillian Gallagher, Stephanie Bertrand, William C Baker, Kalajan Lopez Mercado, Heather Gray-Edwards

J Clin Invest· December 2025

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Intracerebroventricular SPAST-AAV9 gene therapy prevents manifestation of symptoms in a mouse model of SPG4 hereditary spastic paraplegia.
Emanuela Piermarini, Shrobona Guha, Liang Qiang, Heather Gray-Edwards, Miguel Sena-Esteves

Mol Ther· November 2025

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Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial.
Florian Eichler, Oguz I Cataltepe, Rrita Daci, Ajit S Puri, Toloo Taghian, Heather L Gray-Edwards

Nat Med· September 2025

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Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice.
Christiano R R Alves, Sabyasachi Das, Vijai Krishnan, Leillani L Ha, Lauren R Fox, Heather L Gray-Edwards

Nat Biomed Eng· September 2025

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Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases.
Pamela Kell, Sonali Mishra, Heather L Gray-Edwards, Douglas R Martin, Angel Gaudioso

Mol Genet Metab· August 2025

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Cerebrospinal Fluid Flow Enhancement (CFE) Increases the Spatial Distribution of Methotrexate after Intracerebroventricular Administration in a Sheep Model.
Hector Ribeiro Benatti, Toloo Taghian, Olivia Mihalek, Sarah Nath, Jillian Gallagher, Heather L Gray-Edwards

Mol Pharm· July 2025

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AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial.
Connor J Lewis, Precilla D'Souza, Jean M Johnston, Maria T Acosta, Cristan Farmer, Heather Gray-Edwards

medRxiv· July 2025

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Corrigendum to "Natural history progression of MRI brain volumetrics in type II late-infantile and juvenile GM1 gangliosidosis patients" [Molecular Genetics and Metabolism 2025 Mar;144(3):109025].
Josephine Kolstad, Christopher Zoppo, Jean M Johnston, Precilla D'Souza, Anna Luisa Kühn, Heather L Gray-Edwards

Mol Genet Metab· May 2025

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Natural history progression of MRI brain volumetrics in type II late-infantile and juvenile GM1 gangliosidosis patients.
Josephine Kolstad, Christopher Zoppo, Jean M Johnston, Precilla D'Souza, Anna Luisa Kühn, Heather L Gray-Edwards

Mol Genet Metab· March 2025

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digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease.
Jiaming Wang, Laura E Poskitt, Jillian Gallagher, Erik G Puffenberger, R Max Wynn, Heather Gray-Edwards

Sci Transl Med· February 2025

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Clinical and biochemical abnormalities in a feline model of GM2 activator deficiency.
Sidney J Beecy, Amanda L Gross, Anne S Maguire, Leah M K Hoffman, Elise B Diffie, Heather L Gray-Edwards

Mol Genet Metab· January 2025

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Quantifying and mitigating motor phenotypes induced by antisense oligonucleotides in the central nervous system.
Michael P Moazami, Julia M Rembetsy-Brown, Samantha L Sarli, Holly R McEachern, Feng Wang, Heather Gray-Edwards

Mol Ther· December 2024

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Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases.
Rrita Daci, Heather Gray-Edwards, Mohammed Salman Shazeeb, Zeynep Vardar, Behroze Vachha

Hum Gene Ther· November 2024

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Intravenous gene therapy improves lifespan and clinical outcomes in feline Sandhoff Disease.
Anne S Maguire, Linh Ta, Amanda L Gross, Devin E Osterhoudt, Jessica S Cannon, Heather L Gray-Edwards

bioRxiv· November 2024

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A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP.
Hector Ribeiro Benatti, Vania Anagnostakou, Toloo Taghian, Erin F Hall, Sarah Nath, Heather L Gray-Edwards

Mol Ther· October 2024

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Quantitative reliability assessment of brain MRI volumetric measurements in type II GM1 gangliosidosis patients.
Christopher Zoppo, Josephine Kolstad, Jean Johnston, Precilla D'Souza, Anna Luisa Kühn, Heather Gray-Edwards

Front Neuroimaging· September 2024

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A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market.
Nathan Yingling, Miguel Sena-Esteves, Heather L Gray-Edwards

Hum Gene Ther· February 2024

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Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model.
Hector Ribeiro Benatti, Rachel D Prestigiacomo, Toloo Taghian, Rachael Miller, Robert King, Heather L Gray-Edwards

Mol Ther Methods Clin Dev· December 2023

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Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy.
Aime K Johnson, Victoria J McCurdy, Heather L Gray-Edwards, Anne S Maguire, J Nicholas Cochran

Ann Neurol· November 2023

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Precision medicine using whole genome sequencing in a cat identifies a novel COL5A1 variant for classical Ehlers-Danlos syndrome.
Abigail McElroy, Heather Gray-Edwards, Lyndon M Coghill, Leslie A Lyons

J Vet Intern Med· November 2023

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Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.
Manuela Corti, Barry J Byrne, Dominic J Gessler, Grace Thompson, Samantha Norman, Heather L Gray-Edwards

Mol Ther Methods Clin Dev· September 2023

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A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis.
Pamela Kell, Rohini Sidhu, Mingxing Qian, Sonali Mishra, Elena-Raluca Nicoli, Heather L Gray-Edwards

EBioMedicine· June 2023

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Encouraging outcomes of stemless ceramic head anatomic shoulder arthroplasty in severe primary osteoarthritis (Walch type B glenoids).
Gray Alexander Edwards, Jennifer Coghlan, Jeff Hughes, Warwick Wright, Richard Dallalana

Shoulder Elbow· February 2023

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Levels of Synaptic Proteins in Brain and Neurofilament Light Chain in Cerebrospinal Fluid and Plasma of OVT73 Huntington's Disease Sheep Support a Prodromal Disease State.
Ellen Sapp, Adel Boudi, Suzanne J Reid, Bianca A Trombetta, Pia Kivisäkk, Heather Gray-Edwards

J Huntingtons Dis· January 2023

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Distal biceps repair through a single incision with the use of a knotless cortical button device: Mid-term results.
Angelos Assiotis, Jonathan French, Gray Edwards, Philip A McCann, Naomi Chalk

Shoulder Elbow· December 2022

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