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Efrain Sanchez‐Ortiz

Observed names: E. Sanchez-Ortiz, Efrain Sanchez-Ortiz, Efrain Sánchez-Ortiz, Efraín Sánchez-Ortiz, Sanchez-Ortiz, Efrain
ORCID: 0000-0001-9358-1630
Observed ORCID: 0000-0001-9358-1630
OpenAlex ID: A5085694146
Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
2015 · Chengzu Long, Leonela Amoasii, et al. · Science · 940
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
2018 · Leonela Amoasii, John C. W. Hildyard, et al. · Science · 546
Control of muscle formation by the fusogenic micropeptide myomixer
2017 · Pengpeng Bi, Andres Ramirez-Martinez, et al. · Science · 398
Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing
2021 · F. Chemello, A. C. Chai, et al. · Science Advances · 249
Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
2017 · Leonela Amoasii, Chengzu Long, et al. · Science Translational Medicine · 247
Dynamic Transcriptional Responses to Injury of Regenerative and Non-regenerative Cardiomyocytes Revealed by Single-Nucleus RNA Sequencing
2020 · Miao Cui, Zhaoning Wang, et al. · Developmental Cell · 177
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Precise genomic editing of pathogenic mutations in RBM20 rescues dilated cardiomyopathy
2022 · Takahiko Nishiyama, Yu Zhang, et al. · Science Translational Medicine · 170
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Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system
2020 · Yu Zhang, Hui Li, et al. · Science Advances · 162
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A Twist2-dependent progenitor cell contributes to adult skeletal muscle
2017 · Ning Liu, Glynnis A. Garry, et al. · Nature Cell Biology · 144
Fusogenic micropeptide Myomixer is essential for satellite cell fusion and muscle regeneration
2018 · Pengpeng Bi, John R. McAnally, et al. · Proceedings of the National Academy of Sciences · 104
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TrkAGene Ablation in Basal Forebrain Results in Dysfunction of the Cholinergic Circuitry
2012 · Efrain Sanchez-Ortiz, Daishi Yui, et al. · Journal of Neuroscience · 97
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Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing
2020 · Yi-Li Min, Francesco Chemello, et al. · Molecular Therapy · 71
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NF1 regulation of RAS/ERK signaling is required for appropriate granule neuron progenitor expansion and migration in cerebellar development
2014 · Efrain Sanchez-Ortiz, Woosung Cho, et al. · Genes & Development · 60
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NURR1 activation in skeletal muscle controls systemic energy homeostasis
2019 · Leonela Amoasii, Efrain Sanchez-Ortiz, et al. · Proceedings of the National Academy of Sciences · 52
A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation
2021 · Yu Zhang, Takahiko Nishiyama, et al. · Molecular Therapy — Methods & Clinical Development · 49
A MED13-dependent skeletal muscle gene program controls systemic glucose homeostasis and hepatic metabolism
2016 · Leonela Amoasii, William Holland, et al. · Genes & Development · 45
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In vivo non-invasive monitoring of dystrophin correction in a new Duchenne muscular dystrophy reporter mouse
2019 · Leonela Amoasii, Hui Li, et al. · Nature Communications · 43
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A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing
2022 · Yu Zhang, Hui Li, et al. · Molecular Therapy — Nucleic Acids · 36
Dynamic Transcriptional Responses to Injury of Regenerative and Non-regenerative Cardiomyocytes Revealed by Single-Nucleus RNA Sequencing
2020 · Miao Cui, Zhaoning Wang, et al. · Developmental Cell · 35
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Single-swap editing for the correction of common Duchenne muscular dystrophy mutations
2023 · Andreas C. Chai, Francesco Chemello, et al. · Molecular Therapy — Nucleic Acids · 31
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Protein phosphatase 5 protects neurons against amyloid‐β toxicity
2009 · Efrain Sanchez‐Ortiz, Byoung Kwon Hahm, et al. · Journal of Neurochemistry · 28
Sema3a-Nrp1 Signaling Mediates Fast-Twitch Myofiber Specificity of Tw2+ Cells
2019 · Stephen Li, Dileep Karri, et al. · Developmental Cell · 23
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Identification of a multipotent Twist2-expressing cell population in the adult heart
2018 · Yi-Li Min, Priscilla Jaichander, et al. · Proceedings of the National Academy of Sciences · 18
Genomic Editing of a Pathogenic Sequence Variant in ACTA2 Rescues Multisystemic Smooth Muscle Dysfunction Syndrome in Mice
2025 · Qianqian Ding, Peiheng Gan, et al. · Circulation · 17
The PD-1–PD-L1 pathway maintains an immunosuppressive environment essential for neonatal heart regeneration
2024 · Stephanie Vargas Aguilar, Miao Cui, et al. · Nature Cardiovascular Research · 16
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Works count: 36
Citation count: 3,777
H-index: 22
I10-index: 26
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