ArticleNature2023
A framework for individualized splice-switching oligonucleotide therapy.
Registry-linked trialOpen access · hybridAbstract read
In one paragraph
Article in Nature, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07215416 (A Phase 1/2 Study of Antisense Oligonucleotide Therapy for Treatment of Ataxia-Telangiectasia), which is not on this map. Cited by 112 papers.
0numbers the graph read from it
0cells of the map it votes in
112citing papers in PubMed
21.5field-weighted citation impact, top 1% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
NCT07215416 phase1 / phase2recruitingnot on this mapstarted 2026, after this paper: background citation
A Phase 1/2 Study of Antisense Oligonucleotide Therapy for Treatment of Ataxia-Telangiectasia
Who cites it
112 citing papers in PubMed, 138 citations in OpenAlex.
- Citron kinase activity controls alternative splicing events that drive prostate cancer growth.Science advances · 2026Article
- Ataxia-Telangiectasia, DNA Repair Deficits, Immune Dysfunction, and Cancer: A Review of Mechanisms, Clinical Presentations, and Emerging Therapies.Clinical reviews in allergy & immunology · 2026Review
- Neurological disease progression in adults with ataxia-telangiectasia: a longitudinal study.Brain : a journal of neurology · 2026Article
- MASQ: Toward clinical quantification of disease-associated alternative splicing.Molecular therapy. Nucleic acids · 2026Article
- Safety and toxicity assessment of antisense oligonucleotides in brain-relevant human cellular models.Molecular therapy. Nucleic acids · 2026Review
- ESRP1-regulated DNMT3B isoform switching determines the malignant potential of pancreatic cancer.Oncogene · 2026Article
- Review
- Recent Advances in Therapy for the Neurodegenerative Disorder Ataxia-Telangiectasia.International journal of molecular sciences · 2026Review
- Engineering the Future of Precision Medicine: A Comprehensive Guide to RNA Therapeutics.Current issues in molecular biology · 2026Review
- Tools and tactics for studying alternative splicing.Nature reviews. Genetics · 2026Review
- Chemical Modification Strategies for Therapeutic Oligonucleotides: Mechanism Compatibility, Design Trade-Offs, and Translational Barriers.Molecules (Basel, Switzerland) · 2026Review
- SNRPD2-CPSF7-UBE2K axis drives ovarian cancer progression via alternative splicing-polyadenylation crosstalk.Oncogene · 2026Article
- The emerging roles of alternative splicing in modulating tumor immune responses and immunotherapies.Cell death and differentiation · 2026Review
- From N-of-1 to versatility in propionic acidemia: Antisense oligonucleotide-mediated skipping of a constitutiveMolecular therapy. Nucleic acids · 2026Article
- Expanding the toolbox: Emerging antisense oligonucleotide mechanisms for modulating gene expression.Molecular therapy. Nucleic acids · 2026Review
- Article
- Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies.JAMA neurology · 2026Article
- Allele-specific antisense oligonucleotide treatment rescuesbioRxiv : the preprint server for biology · 2026Article
- Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies.Orphanet journal of rare diseases · 2026Review
- Enhanced splicing modulation by NMA-modified antisense oligonucleotides.Nucleic acids research · 2026Article
52 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
Authors and funding
29 authors at 4 institutions in 3 countries.
Jinkuk Kim *Graduate School of Medical Science and Engineering, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea. jinkuk@kaist.ac.kr.ORCID 0000-0003-0564-3294
Sijae Woo *Graduate School of Medical Science and Engineering, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea.
Claudio M de Gusmao *Department of Neurology, Boston Children's Hospital, Boston, MA, USA.
Boxun Zhao *Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.ORCID 0000-0003-2337-5756
Diana H ChinDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.ORCID 0000-0002-1645-3552
Renata L DiDonatoDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
Minh A NguyenDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
Tojo NakayamaDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.ORCID 0000-0003-2110-6878
Chunguang April HuDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
Aubrie SoucyDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.ORCID 0000-0001-8633-0032
Ashley KuniholmInstitutional Center for Clinical and Translational Research, Boston Children's Hospital, Boston, MA, USA.
Jennifer Karlin ThorntonAtaxia Telangiectasia Children's Project, Coconut Creek, FL, USA.
Olivia RiccardiDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
Danielle A FriedmanDepartment of Neurology, Boston Children's Hospital, Boston, MA, USA.
Christelle Moufawad El AchkarDepartment of Neurology, Boston Children's Hospital, Boston, MA, USA.
Zane DashDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.ORCID 0009-0002-7536-9622
Laura CornelissenDepartment of Anesthesiology, Critical Care and Pain Medicine, Boston Children's Hospital, Boston, MA, USA.
Carolina DonadoDepartment of Anesthesiology, Critical Care and Pain Medicine, Boston Children's Hospital, Boston, MA, USA.
Kamli N W FaourDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
Lynn W BushDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
Victoria SuslovitchDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
Claudia LentucciDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
Peter J ParkDepartment of Biomedical Informatics, Harvard Medical School, Boston, MA, USA.ORCID 0000-0001-9378-960X
Eunjung Alice LeeDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.ORCID 0000-0002-6574-9261
Al PattersonHarvard Medical School, Boston, MA, USA.
Anthony A PhilippakisEric and Wendy Schmidt Center, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Brad MargusAtaxia Telangiectasia Children's Project, Coconut Creek, FL, USA.ORCID 0009-0009-5447-6630
Charles B BerdeHarvard Medical School, Boston, MA, USA.
Timothy W YuDivision of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA. timothy.yu@childrens.harvard.edu.ORCID 0000-0003-2988-7701
Boston Children's Hospital · USBroad Institute · USKorea Advanced Institute of Science and Technology · KRHarvard University · US
Funding
Harvard Clinical and Translational Science Center (UL1)UL1TR000170 · NCATS · HARVARD MEDICAL SCHOOL · PI NADLER, LEE MARSHALL · 2012 to 2013
$31.4MGenetic Analysis and Manipulation Core (GAEC)P50HD105351 · NICHD · BOSTON CHILDREN'S HOSPITAL · PI SCOTT Loren POMEROY, MUSTAFA SAHIN · 2021 to 2026
$9.4MMechanism for endogenous retroelements to mimic ancient exogenous identities in aging and diseased human tissueDP2AG072437 · NIA · BOSTON CHILDREN'S HOSPITAL · PI LEE, EUNJUNG ALICE · 2020 to 2020
$2.7MNCATS NIH HHS UL1 TR000170NIA NIH HHS DP2 AG072437NICHD NIH HHS P50 HD105351
Abstract
Splice-switching antisense oligonucleotides (ASOs) could be used to treat a subset of individuals with genetic diseases
Indexed as
Ataxia TelangiectasiaRNA SplicingChildExonsHumansIntronsOligonucleotides, AntisensePilot ProjectsPrecision MedicineProspective StudiesWhole Genome SequencingOligonucleotides, Antisense
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY
Read underepoch 390
Registered trials
NCT07215416ClinicalTrials.gov
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.