Results 121 to 130 of about 37,661 (243)

BODIPY‐based J‐aggregates: From supramolecular chemistry to NIR bioimaging and phototherapies

Bulletin of the Korean Chemical Society, EarlyView.
BODIPY dyes form ordered J‐aggregates through controlled supramolecular self‐assembly, producing narrow, red‐shifted absorption bands with small Stokes shifts. This review highlights design strategies governing J‐aggregation, structure–property relationships, and applications in sensing, bioimaging, and phototherapy, guiding functional BODIPY‐based ...
Pooja Sharma, Yeonkyeong Lee, Youngmi Kim   +2 more
wiley   +1 more source

Broadening applications for intrathecal gene therapy: a case for lysosomal storage diseases. [PDF]

EBioMedicine
eBioMedicine.
europepmc   +1 more source

Editorial: Neuronopathic lysosomal storage diseases - specific neuronal characteristics and therapeutic approaches. [PDF]

Front Mol Neurosci, 2022
Flunkert S, Hutter-Paier B, Sun Y, Kehr J.   +3 more
europepmc   +1 more source

Overcoming intranasal delivery barriers with ultrastable polyzwitterionic siRNA nanocages for enhanced glioblastoma therapy

BMEMat, EarlyView.
A ROS‐responsive, zwitterionic nanocage enables stable, intranasal siRNA delivery to glioblastoma, promoting deep tumor penetration via non‐degradative pathways and trigeminal nerve transport. This platform achieves durable gene silencing and tumor suppression, offering a non‐invasive, storage‐stable strategy for treating glioma and other neurological ...
Jingwen Xie, Jingxing Si, Xiaobei Deng, Jing Tan, Ruijie Zhu, Jizhou Qi, Gaoxia Ma, Bin Wang, Yi Wang, Yunzhou Guo, Yuping Chen, Shiqun Shao, Youqing Shen   +12 more
wiley   +1 more source

Japanese experience of newborn screening for lysosomal storage diseases and adrenoleukodystrophy. [PDF]

Orphanet J Rare Dis
Onuki T, Tajika M, Sugiyama Y, Shimura M, Ichimoto K, Tanaka T, Nyuzuki H, Kosuga M, Migita O, Ito T, Sasai H, Bo R, Hamada J, Hamazaki T, Sakai N, Inoue T, Nakamura K, Okuyama T, Murayama K.   +18 more
europepmc   +1 more source

Correction to: A systematic review of economic evaluations of enzyme replacement therapy in Lysosomal storage diseases. [PDF]

Cost Eff Resour Alloc, 2022
Katsigianni EI, Petrou P.
europepmc   +1 more source

Carrier‐free self‐assembled nanomedicine for combination‐therapy of acute myeloid leukemia

BMEMat, EarlyView.
CPDS combines drugs with three different mechanisms of action to achieve a multi‐mechanism combination therapy for AML by directly killing tumor cells and activating anti‐tumor immunity. Abstract As the main acute myeloid leukemia (AML) clinical treatment, the chemotherapy alone cannot meet the clinical therapeutic needs due to the high heterogeneity ...
Meihong Chai, Rui Zhang, Haiyin Yang, Jiyue Xing, Tianyuan Hu, Mengxi Zhu, Rui Li, Xiaoli Chen, Beibei Zhao, Yang Lan, Stefan Barth, Yuanyu Huang, Li Zhang, Mengjie Zhang, Huining He   +14 more
wiley   +1 more source

How to manage hypersensitivity reactions to enzyme replacement therapy in lysosomal storage diseases? [PDF]

Orphanet J Rare Dis
Spataro F, Solimando AG, Desantis V, Vacca A, Di Girolamo A, Ria R.   +5 more
europepmc   +1 more source

Altered blood-brain barrier transport of nanotherapeutics in lysosomal storage diseases. [PDF]

J Control Release, 2022
Solomon M, Loeck M, Silva-Abreu M, Moscoso R, Bautista R, Vigo M, Muro S.   +6 more
europepmc   +1 more source

First National Expanded Genomic Newborn Screening Program in Qatar; A Pilot Study, Doha‐Heidelberg Collaboration

American Journal of Medical Genetics Part A, EarlyView.
Reem Alsulaiman, Sarah Okashah, Radja Messai Badji, Fatemeh Abbaszadeh, Yaser Alsarraj, Rehab Ali Abdulrahman, Noora Shahbeck, Thalib Moideen, Reema El Hassan, Fatima Al‐Hamaydeh, Einas Alkuwari, Said Ismail, Hilal Al‐ Rifai, Ghassan Abdoh, Christian P. Schaaf, Georg Hoffmann, Saad Al‐Kaabi, Wadha Al‐Muftah, Tawfeg Ben‐Omran   +18 more
wiley   +1 more source