Results 91 to 100 of about 2,049,710 (211)

Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC

Disease Models & Mechanisms, 2016
Mucopolysaccharidosis type IIIC (MPSIIIC) is a severe lysosomal storage disease caused by deficiency in activity of the transmembrane enzyme heparan-α-glucosaminide N-acetyltransferase (HGSNAT) that catalyses the N-acetylation of α-glucosamine residues ...
Sara Marcó, Anna Pujol, Carles Roca, Sandra Motas, Albert Ribera, Miguel Garcia, Maria Molas, Pilar Villacampa, Cristian S. Melia, Víctor Sánchez, Xavier Sánchez, Joan Bertolin, Jesús Ruberte, Virginia Haurigot, Fatima Bosch   +14 more
doaj   +1 more source

Chitosan-Hyaluronic acid hybrid vectors for retinal gene therapy [PDF]

, 2012
info:eu-repo/semantics ...
Bitoque, Diogo, Oliveira, Ana, Silva, Gabriela A.   +2 more
core  

Development of convolutional neural networks for automated brain-wide histopathological analysis in mouse models of synucleinopathies

npj Parkinson's Disease
Preclinical animal models are indispensable for uncovering disease mechanisms and developing novel therapeutic interventions in synucleinopathies. Key readouts including neuronal cell death, neuroinflammation and alpha-synuclein protein aggregation, are ...
A. Barber-Janer, E. Van Acker, E. Vonck, D. Plessers, F. Rosada, C. Van den Haute, V. Baekelandt, W. Peelaerts   +7 more
doaj   +1 more source

Corrigendum: Optimized riboswitch-regulated AAV vector for VEGF-B gene therapy

Frontiers in Medicine, 2023
Reetta A. E. Eriksson, Reetta A. E. Eriksson, Tiina Nieminen, Lionel Galibert, Sanna K. Peltola, Petra Tikkanen, Piia Käyhty, Piia Käyhty, Hanna M. Leinonen, Igor Oruetxebarria, Saana Lepola, Anniina J. Valkama, Eevi M. Lipponen, Hanna P. Lesch, Seppo Ylä-Herttuala, Seppo Ylä-Herttuala, Kari J. Airenne   +16 more
doaj   +1 more source

Impact of rapid mecA polymerase chain reaction rapid diagnostic testing for Staphylococcus aureus in a pediatric setting [PDF]

, 2018
Rapid molecular technology can detect the mecA resistance gene in Staphylococcus aureus (SA), predicting methicillin susceptibility in under one hour. In combination with antimicrobial stewardship program interventions in adults with SA bacteremia, rapid
Drwiega, Emily
core   +1 more source

Ewing’s Sarcoma [PDF]

, 2018
Ewing’s sarcoma is the second most common malignant tumor of the bone occurring in children and adolescents. Typically, patients present between the ages of 10 and 20, with the disease having a slight predilection for males.1 Tumors often arise in the ...
Fenderson, PhD, Bruce A., Lucasti, BS, Chris   +1 more
core   +1 more source

Gene therapy [PDF]

Nature, 1992
openaire   +2 more sources

Exploring RNA-targeted gene therapy approaches for hypertrophic cardiomyopathy [PDF]

, 2012
Relatório de projeto no âmbito do Programa de Bolsas Universidade de Lisboa/Fundação Amadeu Dias (2011/2012). Universidade de Lisboa.
Freitas, Ana
core  

Adeno-associated virus as a delivery vector for gene therapy of human diseases

Signal Transduction and Targeted Therapy
Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues.
Jiang-Hui Wang, Dominic J. Gessler, Wei Zhan, Thomas L. Gallagher, Guangping Gao   +4 more
doaj   +1 more source

The Development of Regulatory Standards for Gene Therapy in the European Union [PDF]

, 1994
This note examines the EU\u27s efforts to regulate gene therapy, considering the Union\u27s resolve to establish scientifically, economically, and morally sound parameters acceptable to its varied constituency.
De Jager, Charles F.
core   +1 more source