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Is Exon Skipping a Viable Therapeutic Approach for Vascular Ehlers–Danlos Syndrome with Mutations in COL3A1 Exon 10 or 15?
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- International Journal of Molecular Sciences, 2024, v. 25, n. 16, p. 8816, doi. 10.3390/ijms25168816
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- Article
By-passing the nonsense mutation in the 4<sup> CV</sup> mouse model of muscular dystrophy by induced exon skipping.
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- Journal of Gene Medicine, 2009, v. 11, n. 1, p. 46, doi. 10.1002/jgm.1265
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- Article
Expanding Patient Access to Cancer Care Services: Key results from a national survey show a range of new initiatives.
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- Oncology Issues, 2020, v. 35, n. 6, p. 48, doi. 10.1080/10463356.2020.1826205
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- Article
YAPping about and not forgetting TAZ.
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- FEBS Letters, 2019, v. 593, n. 3, p. 253, doi. 10.1002/1873-3468.13318
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- Article
Regulation of eukaryotic gene expression by the untranslated gene regions and other non-coding elements.
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- Cellular & Molecular Life Sciences, 2012, v. 69, n. 21, p. 3613, doi. 10.1007/s00018-012-0990-9
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- Article
Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era.
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- Medicinal Research Reviews, 2020, v. 40, n. 6, p. 2650, doi. 10.1002/med.21718
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- Article
In Vitro Validation of Phosphorodiamidate Morpholino Oligomers.
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- Molecules, 2019, v. 24, n. 16, p. 2922, doi. 10.3390/molecules24162922
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- Article
The potential of eye-tracking as a sensitive measure of behavioural change in response to intervention.
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- Scientific Reports, 2018, v. 8, n. 1, p. 1, doi. 10.1038/s41598-018-32444-9
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- Article
Measuring the Impact of Bilingualism on Executive Functioning Via Inhibitory Control Abilities in Autistic Children.
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- Journal of Autism & Developmental Disorders, 2022, v. 52, n. 8, p. 3560, doi. 10.1007/s10803-021-05234-y
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- Article
The Impact of Bilingualism on Everyday Executive Functions of English-Arabic Autistic Children: Through a Parent-Teacher Lens.
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- Journal of Autism & Developmental Disorders, 2022, v. 52, n. 5, p. 2224, doi. 10.1007/s10803-021-05114-5
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- Article
An International Survey of Parental Attitudes to Technology Use by Their Autistic Children at Home.
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- Journal of Autism & Developmental Disorders, 2019, v. 49, n. 4, p. 1517, doi. 10.1007/s10803-018-3798-0
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Pros and Cons of Character Portrayals of Autism on TV and Film.
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- Journal of Autism & Developmental Disorders, 2018, v. 48, n. 2, p. 635, doi. 10.1007/s10803-017-3390-z
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- Article
The Role of the Broader Autism Phenotype and Environmental Stressors in the Adjustment of Siblings of Children with Autism Spectrum Disorders in Taiwan and the United Kingdom.
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- Journal of Autism & Developmental Disorders, 2017, v. 47, n. 8, p. 2363, doi. 10.1007/s10803-017-3134-0
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- Article
Brief Report: Young Adults with Autism Spectrum Disorder Show Normal Attention to Eye-Gaze Information—Evidence from a New Change Blindness Paradigm.
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- Journal of Autism & Developmental Disorders, 2008, v. 38, n. 9, p. 1785, doi. 10.1007/s10803-008-0548-8
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- Article
Anti‐ableist language is fully compatible with high‐quality autism research: Response to Singer et al. (2023).
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- Autism Research: Official Journal of the International Society for Autism Research, 2023, v. 16, n. 4, p. 673, doi. 10.1002/aur.2928
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- Article
The Impact of Bilingualism on the Executive Functions of Autistic Children: A Study of English–Arabic Children.
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- Autism Research: Official Journal of the International Society for Autism Research, 2021, v. 14, n. 3, p. 533, doi. 10.1002/aur.2439
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- Article
Autism and Bilingualism: A Qualitative Interview Study of Parents' Perspectives and Experiences.
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- Journal of Speech, Language & Hearing Research, 2017, v. 60, n. 2, p. 435, doi. 10.1044/2016_JSLHR-L-15-0348
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- Article
A Precision Therapy Approach for Retinitis Pigmentosa 11 Using Splice-Switching Antisense Oligonucleotides to Restore the Open Reading Frame of PRPF31.
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- International Journal of Molecular Sciences, 2024, v. 25, n. 6, p. 3391, doi. 10.3390/ijms25063391
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- Article
Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I Fibroblasts.
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- International Journal of Molecular Sciences, 2022, v. 23, n. 7, p. 3937, doi. 10.3390/ijms23073937
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- Article
Proof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52.
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- International Journal of Molecular Sciences, 2021, v. 22, n. 7, p. 3479, doi. 10.3390/ijms22073479
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- Article
Nonsequential Splicing Events Alter Antisense-Mediated Exon Skipping Outcome in COL7A1.
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- International Journal of Molecular Sciences, 2020, v. 21, n. 20, p. 7705, doi. 10.3390/ijms21207705
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- Article
A Splice Intervention Therapy for Autosomal Recessive Juvenile Parkinson's Disease Arising from Parkin Mutations.
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- International Journal of Molecular Sciences, 2020, v. 21, n. 19, p. 7282, doi. 10.3390/ijms21197282
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- Article
Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing.
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- International Journal of Molecular Sciences, 2019, v. 20, n. 21, p. 5434, doi. 10.3390/ijms20215434
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- Article
Systematic Approach to Developing Splice Modulating Antisense Oligonucleotides.
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- International Journal of Molecular Sciences, 2019, v. 20, n. 20, p. 5030, doi. 10.3390/ijms20205030
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- Article
NCLB: The Next Step in the Diminishment of Physical Education and Health.
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- Illinois Journal for Health, Physical Education, Recreation & Dance, 2008, v. 61, p. 58
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- Article
Neurotype-Matching, but Not Being Autistic, Influences Self and Observer Ratings of Interpersonal Rapport.
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- Frontiers in Psychology, 2020, v. 11, p. N.PAG, doi. 10.3389/fpsyg.2020.586171
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- Article
Determinants of Disease Penetrance in PRPF31 -Associated Retinopathy.
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- Genes, 2021, v. 12, n. 10, p. 1542, doi. 10.3390/genes12101542
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- Article
Novel Mutations Found in Individuals with Adult-Onset Pompe Disease.
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- Genes, 2020, v. 11, n. 2, p. 135, doi. 10.3390/genes11020135
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- Article
Pathways to Justice: The Relevance of Holocaust Education in Language Arts Classrooms.
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- Ohio Journal of English Language Arts, 2017, v. 57, n. 1, p. 65
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- Article
Primary over-expression of Aβ PP in muscle does not lead to the development of inclusion body myositis in a new lineage of the MCK-Aβ PP transgenic mouse.
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- International Journal of Experimental Pathology, 2013, v. 94, n. 6, p. 418, doi. 10.1111/iep.12048
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- Article
Limb Girdle Muscular Dystrophy Type 2B (LGMD2B): Diagnosis and Therapeutic Possibilities.
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- International Journal of Molecular Sciences, 2024, v. 25, n. 11, p. 5572, doi. 10.3390/ijms25115572
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- Article
Dystrophin Isoform Induction In Vivo by Antisense-mediated Alternative Splicing.
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- Molecular Therapy, 2010, v. 18, n. 6, p. 1218, doi. 10.1038/mt.2010.45
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- Article
Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping.
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- Molecular Therapy, 2010, v. 18, n. 1, p. 198, doi. 10.1038/mt.2009.248
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- Article
Rational Design of Antisense Oligomers to Induce Dystrophin Exon Skipping.
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- Molecular Therapy, 2009, v. 17, n. 8, p. 1418, doi. 10.1038/mt.2009.49
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- Article
Morpholino Oligomer–Mediated Exon Skipping Averts the Onset of Dystrophic Pathology in the mdx Mouse.
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- Molecular Therapy, 2007, v. 15, n. 9, p. 1587, doi. 10.1038/sj.mt.6300245
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- Article
Co-development of a Best Practice Checklist for Mental Health Data Science: A Delphi Study.
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- Frontiers in Psychiatry, 2021, v. 12, p. 1, doi. 10.3389/fpsyt.2021.643914
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- Article
Creative Thinking in Schools: Finding the "Just Right" Challenge for Students.
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- Gifted Child Today, 2011, v. 34, n. 2, p. 37, doi. 10.1177/107621751103400211
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- Article
TV weather forecasting.
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- Weather (00431656), 2004, v. 59, n. 3, p. 81, doi. 10.1256/wea.172.03
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- Article
Corrigendum: Response to 'Railroading at the FDA'.
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- Nature Biotechnology, 2017, v. 35, n. 5, p. 481, doi. 10.1038/nbt0517-481d
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- Article
Response to 'Railroading at the FDA'.
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- Nature Biotechnology, 2017, v. 35, n. 3, p. 207, doi. 10.1038/nbt.3819
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- Article
Eng 7980: Learning Transfer in History and Theories of Composition.
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- Composition Studies, 2020, v. 48, n. 1, p. 88
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- Article
Associations of Socioeconomic Deprivation and Preterm Birth With Speech, Language, and Communication Concerns Among Children Aged 27 to 30 Months.
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- JAMA Network Open, 2019, v. 2, n. 9, p. e1911027, doi. 10.1001/jamanetworkopen.2019.11027
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- Article
Splice modulating antisense oligonucleotides restore some acid-alpha-glucosidase activity in cells derived from patients with late-onset Pompe disease.
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- Scientific Reports, 2020, v. 10, n. 1, p. 1, doi. 10.1038/s41598-020-63461-2
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- Article
Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides.
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- Scientific Reports, 2019, v. 9, n. 1, p. N.PAG, doi. 10.1038/s41598-019-49385-6
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- Article
Antisense Oligonucleotide Induction of Progerin in Human Myogenic Cells.
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- PLoS ONE, 2014, v. 9, n. 6, p. 1, doi. 10.1371/journal.pone.0098306
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- Article
Revertant Fibers in the <i>mdx</i> Murine Model of Duchenne Muscular Dystrophy: An Age- and Muscle-Related Reappraisal.
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- PLoS ONE, 2013, v. 8, n. 8, p. 1, doi. 10.1371/journal.pone.0072147
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- Article
Improved Antisense Oligonucleotide Design to Suppress Aberrant <i>SMN2</i> Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy.
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- PLoS ONE, 2013, v. 8, n. 4, p. 1, doi. 10.1371/journal.pone.0062114
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- Article
A latent measure explains substantial variance in white matter microstructure across the newborn human brain.
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- Brain Structure & Function, 2017, v. 222, n. 9, p. 4023, doi. 10.1007/s00429-017-1455-6
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- Article
Rapid Detection of Person Information in a Naturalistic Scene.
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- Perception, 2008, v. 37, n. 4, p. 571, doi. 10.1068/p5705
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- Article
Proteomic profiling of antisense-induced exon skipping reveals reversal of pathobiochemical abnormalities in dystrophic mdx diaphragm.
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- Proteomics, 2009, v. 9, n. 3, p. 671, doi. 10.1002/pmic.200800441
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- Article