Results 41 to 50 of about 22,029 (242)

Fusogenic RNA Nanomodules for Fusion‐Mediated and Multiplexed siRNA Delivery

Advanced Functional Materials, EarlyView.
A fusogenic lipid‐layered RNA nanomodules (L‐CRAMs) enable high‐capacity and long‐lasting siRNA delivery through membrane fusion. These nanomodules carry exceptionally large siRNA payloads, avoid conventional endosomal uptake, and release multiple functional siRNAs through Dicer‐mediated processing.
Sunghyun Moon, Iksoo Jang, Taehyeon Kim, Nayoung Yoon, Kyuha Yum, Jong Bum Lee   +5 more
wiley   +1 more source

Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy

Nature Communications, 2021
Mucopolysaccharidosis type IVA (MPSIVA) is a lysosomal storage disorder causing severe skeletal and non-skeletal alterations in patients. Here, the authors generate a MPSIVA rat model that mimics the disabling human pathology and develop an AAV9-Galns ...
Joan Bertolin, Víctor Sánchez, Albert Ribera, Maria Luisa Jaén, Miquel Garcia, Anna Pujol, Xavier Sánchez, Sergio Muñoz, Sara Marcó, Jennifer Pérez, Gemma Elias, Xavier León, Carles Roca, Veronica Jimenez, Pedro Otaegui, Francisca Mulero, Marc Navarro, Jesús Ruberte, Fatima Bosch   +18 more
doaj   +1 more source

Multifunctional Catechol‐Functionalized Cellulose Hydrogels for the Minimally Invasive Treatment of Acute Optic Nerve Injuries

Advanced Healthcare Materials, EarlyView.
Catechol‐functionalized cellulose hydrogels are developed as injectable, bioadhesive platforms for retinal neuroprotection. The hydrogels exhibit tunable rheological and mechanical properties, strong tissue adhesion, and sustained antioxidative activity.
Kai‐Hsiang Chang, Yi‐Ke Lin, Wei‐Li Chen, Ta‐Ching Chen, Jiashing Yu   +4 more
wiley   +1 more source

Nanomaterial Strategies for Pulmonary Delivery of Immunotherapeutics in Lung Cancer Treatment

Advanced Healthcare Materials, EarlyView.
Inhalable immunotherapeutic nanomedicines enable organ‐selective immune modulation by overcoming pulmonary delivery barriers and concentrating therapy within lung tumors. This Review defines how nanomaterial properties govern airway deposition, retention, cellular partitioning, and immune activation across vaccines, checkpoint blockade, STING agonists,
Han Zhang, Wei Tang
wiley   +1 more source

Mitochondria‐Targeted Nanotherapies in Aging Neurodegenerative Disorders: Emerging Prospects and Clinical Potential

Advanced Healthcare Materials, EarlyView.
Mitochondria‐targeted nanotherapies emerge as a promising strategy for combating aging‐associated neurodegenerative disorders (NDs) by restoring mitochondrial function, reducing oxidative stress, and improving neuronal survival. Recent advances in nanotechnology, therapeutic delivery, and translational research are highlighted, providing insights into ...
Dnyandev G. Gadhave, Ashish B. Jadhav, Nitin B. Waghamode, Shubham Khot, Rajiya Khan, Ravi Hole, Shankar M. Dhobale, Manoj Aswar, Keshav Raj Paudel   +8 more
wiley   +1 more source

Recent Advances in Ferrite‐Based Materials for Biomedical Applications: A Comprehensive Review

Advanced Materials, EarlyView.
Ferrite nanoplatforms are presented as tunable biomedical materials in which synthesis control, cation engineering, defect/morphology regulation, and surface functionalization govern structure–property–bioactivity relationships. These design strategies enable multifunctional applications including MRI contrast, magnetic hyperthermia, targeted drug ...
Pramod D. Mhase, Varsha C. Pujari, Akash V. Fulari, Sunil M. Patange, Sean Li, Danyang Wang, Sagar E. Shirsath   +6 more
wiley   +1 more source

Mr BMT Achieves Systemic Macrophage Replacement With Preservation of Tissue Homeostasis

Advanced Science, EarlyView.
Microglia replacement by bone marrow transplantation (Mr BMT) enables systemic replacement of tissue‐resident macrophages. Despite persistent macrophage and tissue remodeling across multiple organs, core biological functions and innate immune responses remain preserved, supporting long‐term maintenance of organismal homeostasis and the therapeutic ...
Yufei Xu, Miaozhan Zou, Yunshang Bai, Yang He, Zhihao Jin, Baozhi Yang, Jinjin Du, Pei Ouyang, Yuanyuan Cai, Yuheng Zhang, Bingying Du, Chengcheng Ge, Jian‐an Gong, Xiaoyu Li, Yuanyi Zheng, Jing Ding, Bo Peng, Yanxia Rao   +17 more
wiley   +1 more source

Lilrb4a Suppression Reprograms Microglia to Mitigate APOE4‐Associated Amyloid Plaques and Cerebral Amyloid Angiopathy in Association With a PPAR‐Linked Pro‐Clearance State

Advanced Science, EarlyView.
Targeting Lilrb4a in Apolipoprotein E4 (APOE4)‐associated Alzheimer's disease (AD) reprograms microglia toward a beneficial, phagocytic state. Genetic deletion or antisense inhibition of Lilrb4a suppresses p‐SHP2/NF‐κB/STAT1 signaling, restores PPAR‐linked lipid and energy metabolism, and reduces amyloid plaque burden and cerebral amyloid angiopathy ...
Changxu Nie, Ruixi Yang, Xiaotong Wang, Ping Jia, Xueqi Zhang, Yaqi Dai, Xue Bai, Sijia Duan, Yufeng Li, Peng Zheng, Xin Tian, Li Jiang, Chao Wang   +12 more
wiley   +1 more source

Dynamic Regulation of Endogenous Transcription Factor Hubs at Single‐Molecule Resolution

Advanced Science, EarlyView.
This study combines single‐molecule microscopy and genome editing to characterize the dynamic behaviors of endogenous oncofusion transcription factor EWS::FLI1 in Ewing sarcoma cells. EWS::FLI1 forms neomorphic hubs that dynamically assemble and dissolve. The hubs are regulated during mitosis, by RNA, and by specific chemicals.
Shawn Yoshida, Yanghao Zhong, Akiko Kumagai, William G. Dunphy, Shasha Chong   +4 more
wiley   +1 more source

Dermatological Characteristics in a Cohort of Patients with Mucopolysaccharidosis from Southwestern Colombia [PDF]

Journal of Inborn Errors of Metabolism and Screening
Background: Mucopolysaccharidoses (MPS) are rare lysosomal storage disorders characterized by glycosaminoglycan accumulation and multisystem involvement, including underreported dermatological features.
Johan Conquett Huertas, Winnie Celorio, Jose Maria Satizabal, Lina Moreno Giraldo   +3 more
doaj   +2 more sources