Sage Journals: Discover world-class research

Retricted access

Meeting report

First published 2025-12

Risdiplam: A Small Molecule mRNA Splice Modifier Approved to Treat SMA Disease

Lutz Müller, Björn Jacobsen, Alexander Nürnberg

Toxicologic Pathology

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Research article

First published 2023-08

TRPM4 Drives Cerebral Edema by Switching to Alternative Splicing Isoform After Experimental Traumatic Brain Injury

Lihan Zhang, Chaojie Li, Yijing He [...]

Journal of Neurotrauma

Free access

Research article

First published 2008-7

Antisense RNA Sequences Modulating the Ataxin-1 Message: Molecular Model of Gene Therapy for Spinocerebellar Ataxia Type 1,a Dominant-Acting Unstable Trinucleotide Repeat Disease

Youxin Gao, Tao Zu, Walter C. Low [...]

Cell Transplantation

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Research article

First published 2005-12

Functional Analysis of 114 Exon-Internal AONs for Targeted DMD Exon Skipping: Indication for Steric Hindrance of SR Protein Binding Sites

Annemieke Aartsma-Rus, Christa L. de Winter, Anneke A.M. Janson [...]

Nucleic Acid Therapeutics

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Research article

First published 1995-02

Human Immunodeficiency Virus Type 2 (HIV-2): Packaging Signal and Associated Negative Regulatory Element

Alfredo Garzino-Demo, Robert C. Gallo, Suresh K. Arya

Human Gene Therapy

Free access

Research article

First published 2014-4

Identification of Agents That Promote Endoplasmic Reticulum Stress Using an Assay That Monitors Luciferase Secretion

Nicole A. Doudican, Shih Ya Wen, Amitabha Mazumder [...]

Journal of Biomolecular Screening

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Research article

First published 2000-04

Modified Human Immunodeficiency Virus-Based Lentiviral Vectors Display Decreased Sensitivity to Trans -Dominant Rev

Mario R. Mautino, W. Jay Ramsey, Jakob Reiser [...]

Human Gene Therapy

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Research article

First published 1999-02

Construction and Characterization of Human Salivary Histatin-5 Multimers

H. Situ, H. Tsai, L.A. Bobek

Journal of Dental Research

Open access

Review article

First published 2023

Implications of Tau Dysregulation in Huntington’s Disease and Potential for New Therapeutics

Isaline Mees, Rebecca M. Nisbet, Anthony J. Hannan [...]

Journal of Huntington's Disease

Open access

Research article

First published 2023

In-silico Structural Modeling of Human Immunodeficiency Virus Proteins

Amir Elalouf

Biomedical Engineering and Computational Biology

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