Miguel Sena‐Esteves

University of Massachusetts Chan Medical School — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

55h-index9.5kcitations222works1.72yr avg
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Research Topics

Virus-based gene therapy research(110), Lysosomal Storage Disorders Research(47), RNA Interference and Gene Delivery(43), Viral Infectious Diseases and Gene Expression in Insects(31), CRISPR and Genetic Engineering(23)

Publications221 total

AAV9 Gene Therapy in Type II GM1 Gangliosidosis — A Phase 1–2 Trial
New England Journal of Medicine·2026
Testing a dual AAV gene therapy vector construct to treat sialidosis and galactosialidosis using small and large animal models
Molecular Genetics and Metabolism·2026
CRISPR-mediated targeting of the LMNA c.745C>T mutation enhances survival and cardiac function in congenital muscular dystrophy
Molecular Therapy Advances·2025Open Access
Intracerebroventricular SPAST-AAV9 gene therapy prevents manifestation of symptoms in a mouse model of SPG4 hereditary spastic paraplegia
Molecular Therapy·2025Open Access
Five-year analysis of efficacy and safety of a bidirectional AAV gene therapy in Tay-Sachs sheep
Journal of Clinical Investigation·2025Open Access
Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial
Nature Medicine·2025· 3 citedOpen Access
AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial
2025· 2 citedOpen Access
AAV gene therapy for Cockayne syndrome
2025
CRISPR/Cas9-mediated elimination of the <i>LMNA</i> c.745C&gt;G pathogenic mutation enhances survival and cardiac function in <i>LMNA</i> -associated congenital muscular dystrophy
2025· 1 citedOpen Access
Intravenous gene therapy improves lifespan and clinical outcomes in feline Sandhoff Disease
2024· 1 citedOpen Access
GM1 gangliosidosis, Morquio disease, galactosialidosis, and sialidosis
Elsevier eBooks·2024
7381 Overexpression of Makorin Ring Finger Protein 3 in the Arcuate Nucleus of Postpubertal Female Mice Impacts Neurokinin B and Kisspeptin Co-Expressing Neurons
Journal of the Endocrine Society·2024Open Access
AAV9-Mediated Intrastriatal Delivery of GNAO1 Reduces Hyperlocomotion in Gnao1 Heterozygous R209H Mutant Mice
Journal of Pharmacology and Experimental Therapeutics·2024Open Access
Focused Ultrasound-Mediated Disruption of the Blood–Brain Barrier for AAV9 Delivery in a Mouse Model of Huntington’s Disease
Pharmaceutics·2024· 11 citedOpen Access
AAV9-mediated Intra-striatal Delivery of GNAO1 Reduces Hyperlocomotion in Gnao1 Heterozygous R209H Mutant Mice
Journal of Pharmacology and Experimental Therapeutics·2024
Expression of ALS-PFN1 impairs vesicular degradation in iPSC-derived microglia
Nature Communications·2024· 18 citedOpen Access
129 Neuroimaging Analysis of Bilateral Thalamic Delivery for Gene Therapy
Neurosurgery·2024
236 Bilateral Intrathalamic Infusion of rAAVrh8-Hexa/Hexb in a Phase 1/2 Clinical Trial for Gene Therapy in Infantile and Juvenile Tay-Sachs and Sandhoff Disease; Surgical Technique
Neurosurgery·2024
EZH2 inhibition reactivates epigenetically silenced FMR1 and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons
Frontiers in Neuroscience·2024Open Access
A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market
Human Gene Therapy·2024· 5 cited
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Frequent Co-authors

Xandra O. Breakefield(11), Heather Gray‐Edwards(9), Douglas R. Martin(9), Casey A. Maguire(7), Neil Aronin(6), Christian Mueller(5), Guangping Gao(5), Thomas N. Seyfried(5), Marian DiFiglia(4), Ellen Sapp(4), Matthew J. Gounis(4), Marike L. D. Broekman(4), Aime K. Johnson(4), Allison M. Bradbury(4), Qin Su(3), Bakhos A. Tannous(3), Gwladys Gernoux(3), Richard P. Moser(3), Sourav Choudhury(3), X. O. Breakefield(3)