Results 241 to 250 of about 156,351 (339)

Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier. [PDF]

Mol Ther, 2023
Critchley BJ, Gaspar HB, Benedetti S.
europepmc   +1 more source

Plasma Proteomics Identifies TAOK3 as a Potential Biomarker of Rheumatoid Arthritis Activity and a Novel Therapeutic Target

Arthritis &Rheumatology, Accepted Article.
Objective Bone destruction associated with active rheumatoid arthritis (RA) remains a major therapeutic challenge, with a lack of reliable molecular markers reflecting bone injury. This study aims to identify novel biomarkers linked to bone destruction in active RA through proteomic analysis, providing new strategies for precise monitoring and targeted
Pengfei Xin, Wenzhen Li, Xinyu A, Jun Shen, Xiaohui Meng, Yanqin Bian, Haihui Han, Zheng Xiang, Lei Ran, Songtao Sun, Jun Xie, Tao Yue, Qi Zhu, Xiaoming Zhang, Lu Xie, Lianbo Xiao   +15 more
wiley   +1 more source

Treatment Beliefs Reflect Unmet Clinical Needs in Lysosomal Storage Diseases: An Opportunity for a Patient-Centered Approach. [PDF]

JIMD Rep
Corazolla EM, Langeveld M, Brands MMMG, Sjouke B, Hollak CEM.   +4 more
europepmc   +1 more source

Selective Screening for Lysosomal Storage Diseases with Dried Blood Spots Collected on Filter Paper in 4,700 High-Risk Colombian Subjects

, 2013
Alfredo Uribe, Roberto Giugliani
openalex   +2 more sources

AMNIOTIC EPITHELIAL CELL IMPLANTATION (AECI) IN LYSOSOMAL STORAGE DISEASES [PDF]

, 1984
Andrew M. Yeager, Hugo W. Moser, Harvey S. Singer, James R. Buck, Reuben Matalon   +4 more
openalex   +1 more source

Optimizing biophysical properties of cellular niches to enhance stem cell‐derived extracellular vesicle function in musculoskeletal regeneration

BMEMat, EarlyView.
Extracellular vesicles (EVs) derived from stem cells show promise for applications in regenerative medicine, but their scalability and yield remain challenges. This review explores the approaches for biophysical modulations within cell niches on EV properties, discusses the current clinical application of EVs, and provides an outlook on the existing ...
Yang Xu, Chung Yin Matthew Cheung, Liling Liu, Hoi Pan Harry Cheung, Kam Yu Tam, Dai Fei Elmer Ker, Sarah H. Cartmell, Chuanbin Mao, Zhiyong Zhang, Dan Michelle Wang   +9 more
wiley   +1 more source

c-Abl/TFEB Pathway Activation as a Common Pathogenic Mechanism in Lysosomal Storage Diseases: Therapeutic Potential of c-Abl Inhibitors. [PDF]

Antioxidants (Basel)
Guerra MV, Castro J, Moreno A, Balboa E, Marugan JJ, Alvarez AR, Zanlungo S.   +6 more
europepmc   +1 more source

The Role of the miR-17-92 Cluster in Autophagy and Atherosclerosis Supports Its Link to Lysosomal Storage Diseases. [PDF]

Cells, 2022
Ortuño-Sahagún D, Enterría-Rosales J, Izquierdo V, Griñán-Ferré C, Pallàs M, González-Castillo C.   +5 more
europepmc   +1 more source

The usage of enzyme replacement treatments, economic burden, and quality of life of patients with four lysosomal storage diseases in Shanghai, China

, 2021
Jiahao Hu, Lin Zhu, Jiangjiang He, Dingguo Li, Qi Kang, Chunlin Jin   +5 more
openalex   +2 more sources

Nanostructured drug delivery systems for posterior segment eye diseases: Strategies to defy ocular barriers

BMEMat, EarlyView.
Efficient drug delivery to the posterior segment of the eye has long been a challenging issue due to the complex ocular barriers. The review focuses on the promise held by nanoplatforms for barrier penetration and the key mechanisms involved, also highlighting their advantages in achieving efficient drug delivery and superior treatment of PSEDs ...
Yifan Shen, Ying Yao, Yan Zhang, Xinyu Shi, Ling Bai, Yang Zhong, Yan Zhang, Kun Zhang   +7 more
wiley   +1 more source