Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of <i>HNRNPH2</i> -related neurodevelopmental disorder

Authors

Ané Korff
Anoop Kavirayani (ORCID: https://orcid.org/0000-0003-3874-3101)
A. Samanta (ORCID: https://orcid.org/0009-0006-9615-606X)
Alfonso Lavado
Joseph Ochaba (ORCID: https://orcid.org/0000-0003-3912-7677)
Tushar Patni (ORCID: https://orcid.org/0000-0003-1605-7207)
Ozan Ozdemir
Yong‐Dong Wang (ORCID: https://orcid.org/0000-0001-8751-9216)
Hong Joo Kim (ORCID: https://orcid.org/0000-0002-9157-1612)
C. Frank Bennett
J. Paul Taylor
Berit Powers (ORCID: https://orcid.org/0009-0001-2001-3752)

Institutions

St. Jude Children's Research Hospital (US)
Ionis Pharmaceuticals (United States) (US)

Publication Details

Journal: Science Translational Medicine
Published: 2026-04-22
DOI: https://doi.org/10.1126/scitranslmed.adx3491
Primary Topic: Genomics and Rare Diseases
Type: article
Field-Weighted Citation Impact: 0.00

Funders

American Lebanese Syrian Associated Charities
National Institutes of Health

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article

Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of HNRNPH2 -related neurodevelopmental disorder

Ané Korff, Anoop Kavirayani, A. Samanta, Alfonso Lavado et al.

Apr 22, 2026

Science Translational Medicine

Genomics and Rare Diseases

article

Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of HNRNPH2 -related neurodevelopmental disorder

Ané Korff, Anoop Kavirayani, A. Samanta, Alfonso Lavado, Joseph Ochaba, Tushar Patni, Ozan Ozdemir, Yong‐Dong Wang, Hong Joo Kim, C. Frank Bennett, J. Paul Taylor, Berit Powers

April 22, 2026article en

Abstract

No abstract available for this paper.

Science Translational MedicineVol. 18(846)

St. Jude Children's Research Hospital (US), Ionis Pharmaceuticals (United States) (US)

American Lebanese Syrian Associated Charities, National Institutes of Health

Openalex Percentile: Top 9%

Genomics and Rare Diseases

DOI Source OpenAlex

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