Results 141 to 150 of about 249,219 (350)

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  

Enzyme Replacement Therapy [PDF]

Clinical Pediatrics, 1997
openaire   +2 more sources

Survival, Quality of Life and Effects of Enzyme Replacement Therapy in Adults with Pompe Disease [PDF]

, 2013
Pompe disease, or glycogen storage disorder type II, is a rare inherited metabolic disorder caused by deficiency of the lysosomal enzyme acid α-glucosidase.
Güngör, D. (Deniz)
core   +1 more source

Versatile Cell Penetrating Peptide for Multimodal CRISPR Gene Editing in Primary Stem Cells

Advanced Functional Materials, EarlyView.
CRISPR machinery in diverse molecular formats (DNA, RNA, and ribonucleic protein) is complexed into nanoparticles with the cell‐friendly arginine‐alanine‐leucine‐alanine (RALA) cell‐penetrating peptide. Nanoparticles are delivered to primary mesenchymal stem cells ex vivo or locally in vivo to facilitate multimodal CRISPR gene editing. This RALA‐CRISPR
Joshua P. Graham, Jose Gabriel Castro, Lisette C. Werba, Joseph Amitrano, Luke Fardone, Kevin P. Francis, Anand Ramamurthi, Michael J. Layden, Helen O. McCarthy, Tomas Gonzalez‐Fernandez   +9 more
wiley   +1 more source

Angiotensin-I Converting Enzyme Genotype-Dependent Benefit from Hormone Replacement Therapy in Isometric Muscle Strength and Bone Mineral Density1 [PDF]

, 2001
David R. Woods, Gladys Onambélé-Pearson, R. C. Woledge, Dawn A. Skelton, Stuart Bruce, Steve E. Humphries, Hugh Montgomery   +6 more
openalex   +1 more source

Enzyme-replacement therapy in life-threatening hypophosphatasia [PDF]

, 2012
et al,, McAlister, William H, Whyte, Michael P   +2 more
core   +2 more sources

Plasma‐Treated Hydrogel for Combined RONS and Chemotherapy Delivery: A Proof‐of‐Concept In Ovo

Advanced Functional Materials, EarlyView.
This study explores plasma‐treated hydrogels (PTH) as a new way to deliver both reactive oxygen and nitrogen species and chemotherapy (Doxorubicin) directly to tumors. In ovo tests show effects after a single dose, especially in osteosarcoma tumors. Tumor weight decrease, and a resistance‐related protein is reduced.
Milica Živanić, Albert Espona‐Noguera, Angela Privat Maldonado, Patrik Matušů, Francesco Tampieri, Maria‐Pau Ginebra, Abraham Lin, Evelien Smits, Annemie Bogaerts, Cristina Canal   +9 more
wiley   +1 more source

Machine Learning‐Enabled Polymer Discovery for Enhanced Pulmonary siRNA Delivery

Advanced Functional Materials, EarlyView.
This study provides an efficient approach to train a machine learning model by merging heterogeneous literature data to predict suitable polymers for siRNA delivery. Without the need for extensive laboratory synthesis, the machine learning enabled a virtual screening and successfully predicted a polymer that is validated for effective gene silencing in
Felix Sieber‐Schäfer, Min Jiang, Adrian Kromer, Anny Nguyen, Müge Molbay, Simone Pinto Carneiro, David Jürgens, Gerald Burgstaller, Bastian Popper, Benjamin Winkeljann, Olivia Monika Merkel   +10 more
wiley   +1 more source

Lung involvement and enzyme replacement therapy in Gaucher's disease [PDF]

, 2001
Orly Goitein
openalex   +1 more source

Evolving Platinum‐Copper Nanostructures for Enhanced Photothermal Therapy and Controlled Copper Release in Cancer Therapy

Advanced Functional Materials, EarlyView.
Platinum‐Copper bimetallic nanoparticles may evolve depending on their synthesis under tumor microenvironment conditions and play a dual role as chemotherapeutic agents after releasing Cu ions and as near infrared photothermal agents with the Pt‐rich remaining frames.
Jose I. Garcia‐Peiro, María Sancho‐Albero, Silvia Miguel, Andrea Mosseri, Felipe Hornos, Rafael Contreras‐Montoya, Jose L. Hueso, Jesus Santamaria   +7 more
wiley   +1 more source