The breakthrough in human induced pluripotent stem cells (hiPSCs) has revolutionized the field of biomedical and pharmaceutical research and opened up vast opportunities for drug discovery and regenerative medicine, especially when combined with gene-editing technology. Numerous healthy and patient-derived hiPSCs for human disease modeling have bee...
Published in Cureus
Background Hepatitis C virus (HCV) is a single-stranded RNA virus, which is frequently transmitted through blood transfusions, contact with infected blood or blood products, and vertical transmission. Injectable drug abusers and transplant recipients are predisposed to HCV infection. It causes acute hepatitis, which may progress to chronic hepatiti...
Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the blood and immune systems has placed them at the forefront of cell and gene therapy development. Recent advances in genome-editing tools, in particular for clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) an...
Aptamers are single-stranded DNA or RNA sequences that bind target molecules with high specificity and affinity. Aptamers exhibit several notable advantages over protein-based therapeutics. Aptamers are non-immunogenic, easier to synthesize and modify, and can bind targets with greater affinity. Due to these benefits, aptamers are considered a prom...
Published in Human gene therapy
With conventional treatments for primary immunodeficiency diseases (PIDs), such as allogeneic stem cell transplantation or autologous gene therapy, still facing important challenges, the rapid development of genome editing technologies to more accurately correct the mutations underlying the onset of genetic disorders has provided a new alternative,...
Published in Brain sciences
Young individuals with sickle cell anaemia (SCA) experience sleep disturbances and often experience daytime tiredness, which in turn may impact on their daytime functioning and academic attainment, but there are few longitudinal data. Data on sleep habits and behaviour were taken on the same day as an in-hospital polysomnography. This study assesse...
Published in Patterns
Many disorders are characterized by more than one aberrant biomarker. When the number of such biomarkers increases, it becomes difficult to define meaningful variation within patients as well as to identify targets for therapeutics. With such complexity and biological uncertainty, traditional techniques are ineffective. We suggest an empirical-dyna...
Published in Nursing children and young people
Sickle cell disease, the most common inherited disorder at birth in the UK, has been included in the UK newborn screening programme since 2006. For parents, receiving the news that their newborn has a serious long-term condition can trigger reactions such as shock, disbelief and guilt. Guidelines on sickle cell disease provide clear screening pathw...
Published in Journal of cellular physiology
Nonhematopoietic stem cells as a delivery platform of therapeutic useful genes have attracted widespread attention in recent years, owing to gained a long lifespan, easy separation, high proliferation, and high transfection capacity. Mesenchymal stem/stromal cells (MSCs) are the choice of the cells for gene and cell therapy due to high self-renewal...
Published in Journal of blood medicine
Factor XIII deficiency may be inherited or acquired. Inherited deficiency is associated with signs and symptoms of minor bleeding from a young age, and possible major bleeding complications, in particular during pregnancy. On the other hand, acquired factor XIII deficiency is usually associated with severe symptoms of major bleeding, in particular ...
