Results 221 to 230 of about 127,002 (321)

RNA‐Based Therapies for Inherited Metabolic Disorders

Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.
ABSTRACT Inherited metabolic disorders (IMDs) are a diverse and complex group of genetic conditions resulting from deficiencies in enzymes, transporters, or cofactors. These deficiencies lead to metabolic dysfunction and severe clinical consequences. Despite significant progress in understanding their molecular basis, treatment options remain limited ...
Reddy Sreekanth Vootukuri, Sonam Gurung, Roopkatha Ghosh, Philippa B. Mills, Julien Baruteau, Haiyan Zhou   +5 more
wiley   +1 more source

Expansion of Splice-Switching Therapy with Antisense Oligonucleotides. [PDF]

Int J Mol Sci
Takeshima Y.
europepmc   +1 more source

RNA‐Binding Proteins and Ferroptosis in Cancer: Mechanism and Therapeutic Implications

MedComm – Oncology, Volume 5, Issue 1, March 2026.
Ferroptosis critically influences cancer cell fate and represents a promising therapeutic strategy. Emerging evidence identifies RNA‐binding proteins (RBPs) as key post‐transcriptional regulators of ferroptosis. The figure summarizes ferroptosis‐related RBPs across cancers: blue RBPs act as tumor suppressors by promoting ferroptosis, whereas red RBPs ...
Linlin Chang, Bin Lu, Haotian Shang, Wanjing Ding, Chunhua Wei, Xiaotian Qi, Pengxing He   +6 more
wiley   +1 more source

Single Antisense Oligonucleotides Correct Diverse Splicing Mutations in Hotspot Exons. [PDF]

Proc Natl Acad Sci U S A
Duan C, Rong S, Buerer L, Neil CR, Zhong Y, Lyu Z, Savatt JM, Strande NT, Fairbrother WG.   +8 more
europepmc   +1 more source

Gene therapy for epilepsy: An emerging, promising approach for a serious neurological disorder

Journal of Internal Medicine, Volume 299, Issue 3, Page 302-327, March 2026.
Abstract Gene therapy is emerging as a groundbreaking strategy for treating epilepsy, offering new hope to patients who do not respond to conventional medications. Despite advancements in anti‐seizure treatments, nearly 30%–40% of individuals with epilepsy continue to experience uncontrolled seizures, highlighting the urgent need for more effective and
Marco Ledri, Merab Kokaia
wiley   +1 more source

SNA-modified antisense oligonucleotides: A new pathway for renal targeting? [PDF]

Mol Ther Nucleic Acids
Dumoulin B, Yamada K, Susztak K.
europepmc   +1 more source

Human Dorsal Root Ganglia Neuronal Cell Line to Study Nociceptive Signaling: A New Pipeline for Pain Therapy

The FASEB Journal, Volume 40, Issue 3, 15 February 2026.
The nociceptive properties of differentiated human HD10.6 cells were characterized by multiple approaches (A). Peripheral sensitization was induced within HD10.6 cells in response to an inflammatory cocktail (B), modeling nociceptors in a chronic pain state.
Sara A. Dochnal, Yixing Du, Daniella Bandari, Kaue Franco Malange, Jack Bryant, Julia Borges Paes Lemes, Abby Whitford, Anna R. Cliffe, Prashant Mali, Kim Dore, Yury I. Miller, Tony L. Yaksh   +11 more
wiley   +1 more source

Tofersen and other antisense oligonucleotides in ALS. [PDF]

Ther Adv Neurol Disord
Ludolph A, Wiesenfarth M.
europepmc   +1 more source

Synthesis, Biological Activity, and Molecular Dynamics Simulations of LNA-Charge Neutral Linkages for Enhanced Splice-Switching Antisense Oligonucleotides. [PDF]

Angew Chem Int Ed Engl
Kennett A, Lie L, Flerin M, Kurt BZ, Baker YR, Hill AC, Ramesh A, Wood MJA, Dhara D, El-Sagheer AH, Duarte F, Brown T.   +11 more
europepmc   +1 more source

Interplay between RNA‐protein interactions and RNA structures in gene regulation

FEBS Open Bio, Volume 16, Issue 2, Page 299-313, February 2026.
Methodological advances in mapping transcriptome‐wide RNA‐protein interactions and RNA structures have started to uncover the potential of RNP conformations in gene regulation. Competing RNA–RNA, RNA‐protein and protein–protein interactions shape the compaction and function of RNPs throughout their lifetime and may provide novel therapeutic targets in ...
Jenni Rapakko, Mauro Scaravilli, Minna‐Liisa Änkö   +2 more
wiley   +1 more source