Comparison of siRNA-mediated silencing of glycosaminoglycan synthesis genes and enzyme replacement therapy for mucopolysaccharidosis in cell culture studies [PDF]
Cytotoxicity of laronidase (Aldurazyme®), employed in enzyme replacement therapy (ERT) for mucopolysaccharidosis type I (MPS I) and various siRNAs, tested previously in studies on substrate reduction therapy (SRT) for mucopolysaccharidoses, was ...
Banecka-Majkutewicz, Zyta +5 more
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Exploiting natural and induced genetic variation to study hematopoiesis [PDF]
PUZZLING WITH DNA Blood cell formation can be studied by making use of natural genetic variation across mouse strains. There are, for example, two mouse strains that do not only differ in fur color, but also in average life span and more specifically in ...
Gerrits, Alice,, Gerrits, Alice
core
Coagulation-balance gene predictors influencing visual prognosis in patients treated with photodynamic therapy for classic choroidal neovascularization secondary to age-related macular degeneration. [PDF]
PURPOSE: To determine whether different coagulation-balance genetic polymorphisms explain the variable clinical outcomes of photodynamic therapy with verteporfin (PDT-V) in Caucasian patients with classic or predominantly classic choroidal ...
Romano, Mario
core
Just Genetics: How far does the moratorium on the use of genetic data by insurers within the UK accord with Rawls’ principles of justice? [PDF]
Since the completion of the Human Genome Project in 2004, understanding of genetic information has expanded, opening much greater possibilities with regard to identifying, diagnosing and treating genetic conditions.
Douglass, Benjamin
core
As research on the unique effects of space environment, especially microgravity and cosmic radiation, on the cardiovascular system is being conducted, research on the development of cardiovascular therapeutics using the space environment is attracting
Hyelim Park, Kyu-Sung Kim
doaj +1 more source
Lung cancer is a complex disease with varying subtypes. The genetic architectures and risk factors that are similar or distinct among these subtypes remain unclear.
Haiwei Wang +4 more
doaj +1 more source
Foreword -- Introduction -- Ch. 1. Understanding and manipulating genes -- 1. Understanding heredity / George W. Burns -- 2. From the discovery of genes to the mapping of genomes / Dinshaw Dadachanji -- 3. Discovering the structure of DNA / James D.
Anderson, W. French
core
Rationally and in silico guided APOBEC3F-directed CBE for enhanced PDAC genetic therapy. [PDF]
Fang Q +12 more
europepmc +1 more source
In utero genetic therapy: Treatment of early onset neurological disorders before they start. [PDF]
Borges B +3 more
europepmc +1 more source
Pharmacological treatment and BBB-targeted genetic therapy for MCT8-dependent hypomyelination in zebrafish. [PDF]
Zada D +3 more
europepmc +1 more source

