ArticleScience translational medicine2021
Antisense oligonucleotide therapy in a humanized mouse model of
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Article in Science translational medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers.
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0cells of the map it votes in
36citing papers in PubMed
7.4field-weighted citation impact, top 2% of its field
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Who cites it
36 citing papers in PubMed, 58 citations in OpenAlex.
- RNA-Based Therapeutics in Genetic Neurodevelopmental Disorders: Bridging Molecular Genetics and Precision Medicine.International journal of molecular sciences · 2026Review
- Fatty-acid-based antimiR-23b delivery in the DMSXL model: A potential therapeutic strategy for brain dysfunction in myotonic dystrophy type 1.Cell reports. Medicine · 2026Article
- Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality in an MECP2 duplication mouse model.Nature communications · 2026Article
- MeCP2 gene dosage-dependent neurodevelopmentally restricted defects arise by aberrant activation of cell fate-determining bivalent genes.Nature communications · 2026Article
- Modulating alternative splicing ofScience translational medicine · 2026Article
- Recent advances in epigenetic therapeutics for Rett syndrome: from mechanisms to clinical trials.Frontiers in behavioral neuroscience · 2026Review
- MECP2 Duplication Uncouples Mitochondrial and Purine Metabolism During neuronal maturation.bioRxiv : the preprint server for biology · 2025Article
- Antisense oligonucleotide therapy mitigates autosomal dominant progressive hearing loss in a murine model of human DFNA2.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Immunomodulation of behavior impairment via spleen-specific targeting lipid nanoparticles in a MeCP2 transgenic mouse model.Scientific reports · 2025Article
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- Comprehensive assessment reveals numerous clinical and neurophysiological differences between MECP2-allelic disorders.Annals of clinical and translational neurology · 2025Article
- An RNA editing strategy rescues gene duplication in a mouse model of MECP2 duplication syndrome and nonhuman primates.Nature neuroscience · 2025Article
- RNA-based therapies for neurodevelopmental disorders: innovative tools for molecular correction.Frontiers in molecular biosciences · 2025Review
- Article
- Epigenetic Regulation and Neurodevelopmental Disorders: From MeCP2 to the TCF20/PHF14 Complex.Genes · 2024Review
- Structural variant allelic heterogeneity in MECP2 duplication syndrome provides insight into clinical severity and variability of disease expression.Genome medicine · 2024Article
- Modeling antisense oligonucleotide therapy in MECP2 duplication syndrome human iPSC-derived neurons reveals gene expression programs responsive to MeCP2 levels.Human molecular genetics · 2024Article
- An Irak1-Mecp2 tandem duplication mouse model for the study of MECP2 duplication syndrome.Disease models & mechanisms · 2024Article
- Development and validation of parent-reported gastrointestinal health scale in MECP2 duplication syndrome.Orphanet journal of rare diseases · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 3 institutions in 1 country.
Yingyao ShaoDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0001-6833-2034
Yehezkel SztainbergDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0001-8647-7341
Qi WangJan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
Sameer S BajikarDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.ORCID 0000-0002-8868-881X
Alexander J TrostleJan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
Ying-Wooi WanDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
Paymaan Jafar-NejadIonis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.
Frank RigoIonis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.
Zhandong LiuJan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.ORCID 0000-0002-7608-0831
Jianrong TangJan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.ORCID 0000-0001-5664-2692
Huda Y ZoghbiDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA. hzoghbi@bcm.edu.ORCID 0000-0002-0700-3349
Baylor College of Medicine · USIonis Pharmaceuticals (United States) · USHoward Hughes Medical Institute · US
Funding
Preclinical and Clincial OutcomesP50HD103555 · NICHD · BAYLOR COLLEGE OF MEDICINE · PI MIRJANA MALETIC-SAVATIC · 2020 to 2026
$9.9MSteps towards a paternal gene activation therapy for Angelman syndromeU54HD083092 · NICHD · BAYLOR COLLEGE OF MEDICINE · PI NELSON, DAVID LOREN, NEUL, JEFFREY L · 2014 to 2019
$7.8MMOLECULAR PATHOGENESIS STUDIES OF RETT SYNDROMER01NS057819 · NINDS · BAYLOR COLLEGE OF MEDICINE · PI ZOGHBI, HUDA Y · 2006 to 2025
$7.5MStudying the effects and mechanisms of deep brain stimulation in Rett syndrome miceR01NS100738 · NINDS · BAYLOR COLLEGE OF MEDICINE · PI Jianrong Tang · 2018 to 2026
$3.3MImaging Mass Spectrometry for metabolome mappingR01GM120033 · NIGMS · BAYLOR COLLEGE OF MEDICINE · PI LIU, ZHANDONG, MALETIC-SAVATIC, MIRJANA · 2017 to 2021
$2.5MDeep brain stimulation in Rett syndrome mice: cognitive benefits and their mechanismsR56NS100738 · NINDS · BAYLOR COLLEGE OF MEDICINE · PI TANG, JIANRONG · 2017 to 2017
$338kInvestigating the role of Growth-differentiation factor 11 in neurodevelopmental and MECP2-related disordersF32HD100048 · NICHD · BAYLOR COLLEGE OF MEDICINE · PI BAJIKAR, SAMEER · 2019 to 2021
$168kNICHD NIH HHS F32 HD100048NICHD NIH HHS P50 HD103555NICHD NIH HHS U54 HD083092NIGMS NIH HHS R01 GM120033NINDS NIH HHS R01 NS057819NINDS NIH HHS R01 NS100738NINDS NIH HHS R56 NS100738
Abstract
Many intellectual disability disorders are due to copy number variations, and, to date, there have been no treatment options tested for this class of diseases.
Indexed as
Methyl-CpG-Binding Protein 2X-Linked Intellectual DisabilityAnimalsDNA Copy Number VariationsMiceOligonucleotides, AntisenseMecp2 protein, mouseMethyl-CpG-Binding Protein 2Oligonucleotides, Antisense
Identifiers
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.