Results 161 to 170 of about 532,411 (388)

Monocyte LOXHD1 and RHOB Expression Predictive of Progressive Systemic Sclerosis–Associated Interstitial Lung Disease

Arthritis Care &Research, EarlyView.
Objective A leading cause of death among patients with scleroderma (SSc), interstitial lung disease (ILD) remains challenging to prognosticate. The discovery of biomarkers that accurately determine which patients would benefit from close monitoring and aggressive therapy would be an essential clinical tool.
Cristina M. Padilla, Robert Lafyatis, Kevin F. Gibson, Christina Morse, Eleanor Valenzi, Xiaoping Chen, Xiaoyun Li, Yanwu Zhao, Yongseok Park, Dana Ascherman, Jonathan Minden, Robyn Domsic, Yingze Zhang, Daniel J. Kass   +13 more
wiley   +1 more source

FIVE-YEARS FOLLOW-UP OF THE RESPIRATORY AND MOTOR INVOLVEMENTS IN PATIENTS WITH LATE ONSET POMPE DISEASE (LOPD) ON IRREGULAR ENZYME REPLACEMENT THERAPY (ERT) [PDF]

, 2020
Nuno Sousa
openalex   +1 more source

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, EarlyView.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy

Drug Design, Development and Therapy, 2016
Ozlem Goker-Alpan,1 Nicola Longo,2 Marie McDonald,3 Suma P Shankar,4,5 Raphael Schiffmann,6 Peter Chang,7 Yinghua Shen,7 Arian Pano7 1Lysosomal Disorders Unit, Fairfax, VA, 2University of Utah, Salt Lake City, UT, 3Department of Pediatrics, Duke ...
Goker-Alpan O, Longo N, McDonald M, Shankar SP, Schiffmann R, Chang P, Shen Y, Pano A   +7 more
doaj  

FeDSNP‐Pa Nanoassemblies: A Triple‐Action Therapeutic Strategy Targeting Oxidative Stress, Inflammation, and Pyroptosis for Retinal Ganglion Cell Protection in Glaucoma

Advanced Functional Materials, EarlyView.
FeDSNP‐Pa, a metallized nanoparticle loaded with sodium pyruvate (Pa), exerts triple therapeutic effects by scavenging reactive oxygen species (ROS), suppressing inflammatory responses, and inhibiting pyroptosis signaling pathways. This multifunctional neuroprotective strategy protecting retinal ganglion cells (RGCs) from elevated intraocular pressure ...
Yukun Wu, Xue Li, Tong Wu, Dan Ji, Yugang Bai, Xuezhi Zhou   +5 more
wiley   +1 more source

3D Multicellular Scaffold Based Model for Advancing Bone Disorder Research

Advanced Functional Materials, EarlyView.
A scalable 3D multicellular in vitro bone model engineered by integrating osteoblasts, osteoclasts, and endothelial cells on biodegradable scaffolds. The system recapitulates key features of human bone remodeling and disease pathology. As a proof of concept, the model mimics osteogenesis imperfecta, demonstrating its potential as a physiologically ...
Gali Guterman‐Ram, Majd Machour, Janette Zavin, Roy Meretzki, Reut Lev‐Ari, Shulamit Levenberg   +5 more
wiley   +1 more source

Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II

Molecular Therapy: Methods & Clinical Development, 2021
Joo-Hyun Seo, M. Kosuga, T. Hamazaki, H. Shintaku, T. Okuyama   +4 more
semanticscholar   +1 more source

General practice [PDF]

, 2004
The remarkable rate of development of medical knowledge and pharmacology affects all medical specialties and in particular general practice, since the latter embraces various aspects of different medical fields. Certain areas of medical practice, such as
Abela, Jurgen C.
core  

Mechanically Tunable Bone Scaffolds: In Vivo Hardening of 3D‐Printed Calcium Phosphate/Polycaprolactone Inks

Advanced Functional Materials, EarlyView.
A 3D bone scaffold with osteogenic properties and capable of hardening in vivo is developed. The scaffold is implanted in a ductile state, and a phase transformation of the ceramic induces the stiffening and strengthening of the scaffold in vivo. Abstract Calcium phosphate 3D printing has revolutionized customized bone grafting.
Miguel Mateu‐Sanz, Pablo Varela, Laura del‐Mazo‐Barbara, Irene Lodoso‐Torrecilla, Emilio Jiménez‐Piqué, Jordi Franch, Miguel Alaminos, Maria‐Pau Ginebra   +7 more
wiley   +1 more source

Enzyme Replacement Therapy [PDF]

Clinical Pediatrics, 1997
openaire   +1 more source