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Miguel Sena-Esteves

Associate Professor

University of Massachusetts Chan Medical School

United States

Research Interests

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Articles (20)

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2026

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2023

Collaborators (11)

Eva-Maria Ratai

Associate Professor

MGH Institute of Health Professions

UNITED STATES

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Terence Flotte

Dean, Provost and Executive Deputy Chancellor

University of Massachusetts Chan Medical School

UNITED STATES

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Ana Rita Batista

University of Massachusetts Chan Medical School

UNITED STATES

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Allison Keeler-Klunk

Assistant Professor

University of Massachusetts Chan Medical School

UNITED STATES

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Heather Gray-Edwards

University of Massachusetts Chan Medical School

UNITED STATES

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Allison Bradbury

Principal Investigator II

Ohio State University

UNITED STATES

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Vania Anagnostakou

University of Massachusetts Chan Medical School

UNITED STATES

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Douglas R. Martin

UNITED STATES

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Caryl E. Sortwell

UNITED STATES

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Joseph Patterson

Assistant Professor

Michigan State University

UNITED STATES

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Maninder Sandey

Anatomic Pathologist/Assistant Professor

Auburn University

UNITED STATES

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Miguel Sena-Esteves

Research Interests

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Articles (20)

Intravenous gene therapy improves lifespan and clinical outcomes in a feline model of Sandhoff disease

AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial

Five-year analysis of efficacy and safety of a bidirectional AAV gene therapy in Tay-Sachs sheep

Intracerebroventricular<i> SPAST</i>-AAV9 gene therapy prevents manifestation of symptoms in a mouse model of<i> SPG4</i> hereditary spastic paraplegia

Testing a dual AAV gene therapy vector construct to treat sialidosis and galactosialidosis using small and large animal models

Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial.

CRISPR/Cas9-mediated elimination of the LMNA c.745C>G pathogenic mutation enhances survival and cardiac function in LMNA-associated congenital muscular dystrophy

Five-year analysis of efficacy and safety ofa bidirectional AAV gene therapy in Tay-Sachs sheep

AAV9-Mediated Intrastriatal Delivery of GNAO1 Reduces Hyperlocomotion in Gnao1 Heterozygous R209H Mutant Mice

Focused Ultrasound-Mediated Disruption of the Blood–Brain Barrier for AAV9 Delivery in a Mouse Model of Huntington’s Disease

A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market

Adeno-associated viral gene therapy for sialidosis using small and large animal models

EZH2 inhibition reactivates epigenetically silenced <i>FMR1</i> and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons

Expression of ALS-PFN1 impairs vesicular degradation in iPSC-derived microglia

Intravenous gene therapy improves lifespan and clinical outcomes in feline Sandhoff Disease

Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy.

A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis

Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model

From mouse to sheep: Generating a sheep model and developing a gene therapy for sialidosis

TREATMENT OF DIFFUSE INTRINSIC PONTINE GLIOMA USING ALLELE-SPECIFIC SIRNA TARGETING H3.3 K27M MUTANT HISTONE

Collaborators (11)

Eva-Maria Ratai

Terence Flotte

Ana Rita Batista

Allison Keeler-Klunk

Heather Gray-Edwards

Allison Bradbury

Vania Anagnostakou

Douglas R. Martin

Caryl E. Sortwell

Joseph Patterson

Maninder Sandey

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