Results 211 to 220 of about 94,605 (281)

Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington's disease. [PDF]

Transl Neurodegener
Ou K, Jia Q, Li D, Li S, Li XJ, Yin P.
europepmc   +1 more source

Biomaterial Strategies for Targeted Intracellular Delivery to Phagocytes

Advanced Functional Materials, Volume 36, Issue 1, 2 January 2026.
Phagocytes are essential to a functional immune system, and their behavior defines disease outcomes. Engineered particles offer a strategic opportunity to target phagocytes, harnessing inflammatory modulation in disease. By tuning features like size, shape, and surface, these systems can modulate immune responses and improve targeted treatment for a ...
Kaitlyn E. Woodworth, Neal I. Callaghan, Locke Davenport Huyer   +2 more
wiley   +1 more source

A Case Study of Model-Informed Drug Development of a Novel PCSK9 Antisense Oligonucleotide. Part 2: Phase 2 to Phase 3. [PDF]

CPT Pharmacometrics Syst Pharmacol
Knöchel J, Nilsson C, Carlsson B, Hofherr A, Johanson P, Rydén-Bergsten T, Hamrén B, Rekić D.   +7 more
europepmc   +1 more source

Prickle4 Drives Microenvironmental Remodeling and Resistance to Parp Inhibition in IDH‐Mutant Glioma

Advanced Science, Volume 13, Issue 3, 14 January 2026.
IDH‐mutant gliomas exhibit sensitivity to PARP inhibitors (PARPi) owing to oncometabolite 2‐HG‐induced DNA repair defects. PARPi treatment induces resistance by upregulating Prickle4, which promotes VEGF‐driven angiogenesis. Combining PARPi veliparib with anti‐angiogenic lenvatinib blocks this pathway, causing tumor regression and prolonged survival in
Ju Yang, Hua Yang, Yifan Yuan, Chenyang Zhang, Ziwei Fu, Yanyan Chen, Yinghong Xiong, Shuyu Chen, Kexin Ling, Ying Liu, Jason T. Huse, Bo Chen, Timothy A. Chan, Zengxin Qi, Zhao Zhang, Xiuping Liu, Yuxiang Wang   +16 more
wiley   +1 more source

Antisense oligonucleotide therapy for patients with Friedreich's ataxia carrying the c.165+5G>C splicing mutation. [PDF]

Mol Ther Nucleic Acids
Yameogo P, Aguilar S, Prakash TP, Rigo F, Lynch DR, Napierala JS, Napierala M.   +6 more
europepmc   +1 more source

NEAT1 Promotes Epileptogenesis in Tuberous Sclerosis Complex

Advanced Science, Volume 13, Issue 1, 5 January 2026.
The primary neurological manifestations of tuberous sclerosis complex (TSC) are intractable epilepsy and intellectual disability. NEAT1 is differentially expressed in TSC‐related epilepsy and influences neuronal excitability by regulating the PI3K/AKT/mTOR signaling pathway.
Suhui Kuang, Tinghong Liu, Zhirong Wei, Jinshan Xu, Jiayi Han, Jiaqi Wang, Feng Zhai, Yiran Tian, Shuli Liang   +8 more
wiley   +1 more source

AmNA-Modified Antisense Oligonucleotide Targeting MCM8 as a Cancer-Specific Chemosensitizer for Platinum Compounds. [PDF]

Cancer Sci
Uchibori Y, Suekuni M, Kokaji Y, Yoshida K, Kiyono T, Kasahara Y, Fujita M.   +6 more
europepmc   +1 more source

tRNA‐Derived Fragment tRF‐22 Promotes Immunosuppression by Inhibiting HnRNPAB Ubiquitination in Esophageal Squamous Cell Carcinoma

Advanced Science, Volume 13, Issue 1, 5 January 2026.
tRF‐22 fosters an immunosuppressive and pro‐oncogenic tumor microenvironment in ESCC by stabilizing hnRNPAB against TRIM25‐mediated ubiquitination and enhancing TGFβ2 expression, which increases PMN‐MDSCs infiltration and suppresses CD8+ T cells. Targeting tRF‐22 or blocking TGFβ signalling improves anti‐PD1 response, offering a promising therapeutic ...
Ling Pan, Xin Qin, Li Gong, Haining Liu, Bo Cheng, Jing Wang, Yajie Hu, Lingxing Zeng, Yiping Wang, Qingxu Song, Yufeng Cheng   +10 more
wiley   +1 more source

Antisense oligonucleotide treatment in a preterm infant with early-onset SCN2A developmental and epileptic encephalopathy. [PDF]

Nat Med
Wagner M, Berecki G, Fazeli W, Nussbaum C, Flemmer AW, Frizzo S, Heer F, Heinen F, Horton R, Jacotin H, Motel W, Spar B, Klein C, Siegel C, Hübener C, Stöcklein S, Paolini M, Staudt M, Tacke M, Wolff M, Petrou S, Souza M, Borggraefe I.   +22 more
europepmc   +1 more source

Targeted Expansion of Treg Cells to Induce Immune Tolerance after Kidney Transplantation

Advanced Science, Volume 13, Issue 1, 5 January 2026.
A dual‐targeting nanoparticle platform that selectively engages graft stromal cells via CD248 receptors while employing kidney‐ specific promoters (NPHS2) to drive localized IL‐ 2/TGF‐βexpression is developed. This approach achieves three therapeutic objectives:1) intragraft Treg polarization without systemic cytokine exposure;2) reduced dependence on ...
Tong Lu, Hongtao Song, Limin He, Zhihao Hu, Yu Li, Chao Xu, Shaojie Liu, Jun Jiang, Yike Zhou, Zhengxuan Li, Zeyu Li, Keying Zhang, Shuaijun Ma, Ruochen Qi, Shichao Han, Changhong Shi, Weihong Wen, Donghui Han, Weijun Qin   +18 more
wiley   +1 more source