Results 161 to 170 of about 171,175 (241)

N-of-1 investigational study of a novel antisense oligonucleotide drug in CHCHD10-related ALS shows early signs of efficacy. [PDF]

Med
Cousin MA, Dagli AI, Mignon L, Shah JS, Prudencio M, Gendron TF, Jain A, Udine E, van Blitterswijk M, Middlebrooks EH, Donahue MH, Dagher JE, Watzlawik JO, Springer W, Parekh T, McEachin ZT, Petrucelli L, Oskarsson B.   +17 more
europepmc   +1 more source

Epigenetic Regulation of Liver Fibrosis: Mechanisms and Therapeutic Implications

Portal Hypertension &Cirrhosis, EarlyView.
This review synthesizes the epigenetic mechanisms—DNA methylation, histone modifications, and non‐coding RNAs—that drive hepatic stellate cell activation and the progression of liver fibrosis. It further evaluates emerging therapeutic strategies targeting these epigenetic regulators, including DNA methyltransferase and histone deacetylase inhibitors ...
Maryam Jadid Tavaf, Mahboobeh Ebrahimi Varkiani, Saeid Abroun, Pedram Asadi‐Sarabi, Shukoofeh Torabi, Elham Rismani, Mohammad Majd Hammour, Andreas K. Nüssler, Massoud Vosough   +8 more
wiley   +1 more source

Expanding the toolbox: Emerging antisense oligonucleotide mechanisms for modulating gene expression. [PDF]

Mol Ther Nucleic Acids
Trew I, Wilton SD, Cale JM, Aung-Htut M.
europepmc   +1 more source

Genomics and epigenomics of tissue repair: Implications for personalized medicine

Precision Medical Sciences, EarlyView.
Genomic and epigenomic mechanisms govern tissue repair and regeneration through dynamic regulation of inflammation, cell fate, and extracellular matrix remodeling. The integration of multi‐omics, artificial intelligence, and precision regenerative therapies enables biomarker‐driven, personalized approaches to enhance healing and minimize fibrosis ...
Jharna Medhi, Chandrashekar Thalluri, Sarad Pawar Naik Bukke, Piyali Dey, Mallikarjun Vasam, Ananda Kumar Chettupalli, Swetha Kumari Koduru, Gali Avinash Kumar   +7 more
wiley   +1 more source

Chemical and Biological Determinants of PEG‐Lipid Shedding From Therapeutic Nanoparticles

Small, EarlyView.
PEG‐lipids play a central role in governing lipid nanoparticle stability, targeting, and function. However, PEG‐lipids can “shed” from the nanoparticle surface in biological environments, with the extent and kinetics of shedding influenced by chemical and biological factors.
Ethan P. Cisneros, Aadya S. Wijesekera, Lisa R. Volpatti   +2 more
wiley   +1 more source

A Luminescent PEI/Cy5‐Citrate‐Functionalized Carbonated Apatite Nanoplatform for In Vitro Gene Delivery and In Vivo Traceability

Small, EarlyView.
A luminescent PEI/Cy5‐citrate‐functionalized carbonated apatite nanoplatform combines biocompatibility, plasmid delivery, and Cy5‐based traceability. The system enables effective in vitro gene delivery while preserving cell viability and allows real‐time in vivo fluorescence tracking of the nanocarrier.
Laura Garrido Martín, Marta García Gallego, Raquel Fernández Penas, José Manuel Peula García, Jaime Gómez Morales, Houria Boulaiz   +5 more
wiley   +1 more source

Neural Stem Cell-Derived Extracellular Vesicles Improve CNS Delivery and Therapeutic Efficacy of Antisense Oligonucleotide in Amyotrophic Lateral Sclerosis. [PDF]

J Extracell Vesicles
Zheng Y, Ellison TJ, Garrard R, Gao J, Lai X, Lan L, Xie J, Jiang J, Yao Y.   +8 more
europepmc   +1 more source

Programmable Assembly of Higher‐Order DNA Nanostructures from Microbial ssDNA Staple Libraries

Small, EarlyView.
A phage‐free, microbial platform produces programmable circular ssDNA libraries that are enzymatically converted into DNA origami staple pools. Rolling‐circle replication generates sequence‐complex ssDNA with >99% fidelity, enabling assembly of DNA tiles and tubules and supporting nanostructure formation even in crude cellular lysates.
Ming Hung Yen, Mallikarjuna Reddy Kesama, Yancheng Du, Jong Hyun Choi, Kevin V. Solomon   +4 more
wiley   +1 more source

Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies.

JAMA Neurol
Sherrill E, Cheerie D, Beck CJ, Whittle EF, Shafi Y, Chandler NJ, Christodoulou J, Daniel J, Hassell J, Lachgar-Ruiz M, Mulhern S, Scotchman E, Sidhu J, Tedja CF, Chitty LS, Cross JH, Scheffer IE, Zhou H, Yu TW, Chau V, Stephenson SEM, Poduri A, Howell KB, McTague A, Costain G, D'Gama AM, Gene-STEPS Study Group.   +26 more
europepmc   +1 more source

Inhaled Angiopoietin-Like 4 Antisense Oligonucleotide Therapy for Lung Injury and Fibrosis. [PDF]

Adv Sci (Weinh)
Fan H, Tan Y, Zhang J, Liu X, Qu J, Chua D, Cheng HS, Kim JHS, Liu YX, Qin C, Liu Y, Li D, Qiu J, Chi M, Bi M, Zhang Q, Li Y, Motamedi Joibari M, Nilsson SK, Tan NS, Fan Y, Li L.   +21 more
europepmc   +1 more source