Results 51 to 60 of about 36,350 (272)
Arthritis &Rheumatology, EarlyView.Clinical tools that can aid in the diagnostic differentiation of juvenile dermatomyositis from muscular dystrophy.
Jacqueline A. Madison +9 more
wiley +1 more source
Perfusion‐Based Production of rAAV via an Intensified Transient Transfection Process
Biotechnology and Bioengineering, EarlyView.ABSTRACT Increasing demand for recombinant adeno‐associated virus (rAAV)‐based gene therapies necessitates increased manufacturing production. Transient transfection of mammalian cells remains the most commonly used method to produce clinical‐grade rAAVs due to its ease of implementation.
Tam N. T. Nguyen +11 more
wiley +1 more source
Automatic Classification of Neuromuscular Diseases in Children Using Photoacoustic Imaging [PDF]
arXiv, 2022 Neuromuscular diseases (NMDs) cause a significant burden for both healthcare systems and society. They can lead to severe progressive muscle weakness, muscle degeneration, contracture, deformity and progressive disability. The NMDs evaluated in this study often manifest in early childhood. As subtypes of disease, e.g.
arxiv
A synthetic platform for developing recombinant adeno‐associated virus type 8 producer cell lines
Biotechnology Progress, EarlyView.Abstract Recombinant adeno‐associated virus (rAAV) is one of the most widely used viral vectors for gene therapy. It is used in very high doses for the treatment of many diseases, making large‐scale production for clinical applications challenging. We have established a synthetic biology‐based platform to construct stable production cell lines, which ...
Yu‐Chieh Lin +5 more
wiley +1 more source
Preimplantation genetic diagnosis associated to Duchenne muscular dystrophy
Einstein (São Paulo)Duchenne muscular dystrophy is the most common muscle disease found in male children. Currently, there is no effective therapy available for Duchenne muscular dystrophy patients. Therefore, it is essential to make a prenatal diagnosis and provide genetic
Bianca Bianco +3 more
doaj +1 more source
Duchenne muscular dystrophy: current cell therapies
Therapeutic Advances in Neurological Disorders, 2015 Duchenne muscular dystrophy is a genetically determined X-linked disease and the most common, progressive pediatric muscle disorder. For decades, research has been conducted to find an effective therapy.
Dorota Sienkiewicz +4 more
doaj +1 more source
ChemMedChem, EarlyView.This study introduced DCAF11 as an E3 ligase for PROTAC‐mediated histone deacetylase (HDAC) degradation. By replacing cereblon with DCAF11 as recruited E3 ligase, a selective HDAC6 degrader (A6) was transformed into pan‐HDAC degraders. The lead compound, 1j (FF2039), potently degraded HDACs from classes I, IIa, and IIb, showing strong antiproliferative
Felix Feller +5 more
wiley +1 more source
Environmental and Molecular Mutagenesis, EarlyView.Abstract Exposure levels without appreciable human health risk may be determined by dividing a point of departure on a dose–response curve (e.g., benchmark dose) by a composite adjustment factor (AF). An “effect severity” AF (ESAF) is employed in some regulatory contexts.
Barbara L. Parsons +17 more
wiley +1 more source
Two-sample nonparametric test for proportional reversed hazards [PDF]
arXiv, 2022 Several works have been undertaken in the context of proportional reversed hazard rate (PRHR) since last few decades. But any specific statistical methodology for the PRHR hypothesis is absent in the literature. In this paper, a two-sample nonparametric test based on two independent samples is proposed for verifying the PRHR assumption.
arxiv
Therapeutics and Clinical Risk Management, 2017 David Feder,1 Miriam Eva Koch,1 Beniamino Palmieri,2 Fernando Luiz Affonso Fonseca,1 Alzira Alves de Siqueira Carvalho3 1Pharmacology Department, Faculdade de Medicina do ABC, Santo André, São Paulo, Brazil; 2Department of General Surgery ...
Feder D +4 more
doaj