Results 261 to 270 of about 124,170 (327)

Antisense oligonucleotides modulate aberrant inclusion of poison exons in SCN1A-related Dravet syndrome. [PDF]

JCI Insight
Tang S, Stamberger H, Calhoun JD, Weckhuysen S, Carvill GL.   +4 more
europepmc   +1 more source

Skeletal Muscle Membrane Permeability Markers Derived From 31P‐MRS May Reflect Disease Activity in Becker Muscular Dystrophy

NMR in Biomedicine, Volume 38, Issue 11, November 2025.
Biomarkers for muscle disease activity are needed for trials in Becker muscular dystrophy (BMD). We investigated magnesium (Mg2+), phosphodiesters (PDE) and pH from 31P‐MRS; and membrane permeability from random permeable barrier model (RBPM) diffusion as candidates, studying ‘preserved’ and ‘progressing’ muscles in patients with BMD versus controls ...
Esther J. Schrama, Melissa T. Hooijmans, Nienke M. van de Velde, Erik H. Niks, Hermien E. Kan, Donnie Cameron   +5 more
wiley   +1 more source

APP antisense oligonucleotides are effective in rescuing mitochondrial phenotypes in human iPSC-derived trisomy 21 astrocytes. [PDF]

Alzheimers Dement
Thirumalai S, Livesey FJ, Patani R, Hung C.   +3 more
europepmc   +1 more source

Loss of SMN Impairs Osteoblast–Osteoclast Coupling via IGF1–Akt–OPG Axis in Spinal Muscular Atrophy

The FASEB Journal, Volume 39, Issue 20, 31 October 2025.
Loss of SMN impairs osteoblast–osteoclast coupling by disrupting the IGF1–Akt–OPG signaling axis, which leads to defective bone remodeling in spinal muscular atrophy. The graphical abstract highlights this molecular mechanism underlying skeletal pathology in SMA.
Taiyang Xiang, Zijie Zhou, Yaoyao Li, Yinxuan Suo, Jun Dai, Xiaoyan Shi, Xiaozhong Zhou, Lei Sheng   +7 more
wiley   +1 more source

Elevating microRNA levels by targeting biogenesis with steric-blocking antisense oligonucleotides. [PDF]

RNA
Havens MA, Hinrich AJ, Rigo F, Hastings ML.   +3 more
europepmc   +1 more source

MuRF1 Partners With TRIM72 to Impair Insulin Signaling in Skeletal Muscle Cells

The FASEB Journal, Volume 39, Issue 19, 15 October 2025.
Insulin activates IRS1–Akt signaling to promote glucose uptake. Under dexamethasone treatment, MuRF1 stabilizes TRIM72, which decreases IRS1 protein abundance and impairs glucose uptake in skeletal muscle cells. ABSTRACT Muscle RING‐finger protein 1 (MuRF1, gene name: TRIM63) is well known as a critical molecular regulator in skeletal muscle atrophy ...
Ibrahim Musa, Alex Peter Seabright, Jonathan Barlow, Yusuke Nishimura   +3 more
wiley   +1 more source

Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease. [PDF]

Clin Transl Med
Weiss L, Carrer M, Shmara A, Martin A, Yin H, Pal P, Cheng C, Ta L, Boock V, Fazeli Y, Chang M, Paguio M, Lee J, Yu H, Weiss J, Grossman TR, Raben N, Jafar-Nejad P, Kimonis V.   +18 more
europepmc   +1 more source

Recent Applications of Mesoporous Silica Nanoparticles in Gene Therapy

Advanced Healthcare Materials, Volume 14, Issue 26, October 7, 2025.
The review summarizes the synthesis of mesoporous silica nanoparticles (MSNs) with modifiable surface properties, functionalization strategies, mechanism of therapeutic payload release, and current applications in gene therapy, focusing on their capabilities in the targeted delivery of therapeutic nucleic acids, CRISPR‐Cas systems, and other genetic ...
Tamanna Binte Huq, Punnya Anil Kumar Jeeja, Sudip Kumar Dam, Sabya Sachi Das, Juan L. Vivero‐Escoto   +4 more
wiley   +1 more source

Site-blocking antisense oligonucleotides as a mechanism to fine-tune MeCP2 expression. [PDF]

RNA
Vanderplow AM, Dodis GE, Rhee Y, Cikowski JJ, Gonzalez S, Smith ML, Gogliotti RG.   +6 more
europepmc   +1 more source

Suppressing t(4;11) Acute Leukemia by Lipopolymer Nanoparticle Delivery of siRNA Targeting KMT2A::AFF1 with Enhanced Extrahepatic Delivery

Advanced Healthcare Materials, Volume 14, Issue 27, October 20, 2025.
This study introduces a new lipopolymer nanoparticle (LPNP) system that efficiently delivers siRNA to leukemia cells. The LPNPs silence the leukemia fusion gene KMT2A::AFF1, induce apoptosis, and decrease leukemia burden in mice. These results demonstrate the potential of LPNPs as a targeted siRNA therapy for acute lymphoblastic leukemia.
Mohammad Nasrullah, Remant KC, Amarnath Praphakar Rajendran, Saba Abbasi Dezfouli, Cezary Kucharski, Xiaoyan Jiang, Spencer B. Gibson, Joseph Brandwein, Olaf Heidenreich, Hasan Uludağ   +9 more
wiley   +1 more source