Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects

H Li, Y Yang, W Hong, M Huang, M Wu… - Signal transduction and …, 2020 - nature.com
Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …

Pathophysiology of sickle cell disease

P Sundd, MT Gladwin, EM Novelli - Annual review of pathology …, 2019 - annualreviews.org
Since the discovery of sickle cell disease (SCD) in 1910, enormous strides have been made
in the elucidation of the pathogenesis of its protean complications, which has inspired recent …

[HTML][HTML] Global, regional, and national prevalence and mortality burden of sickle cell disease, 2000–2021: a systematic analysis from the Global Burden of Disease …

AM Thomson, TA McHugh, AP Oron, C Teply… - The Lancet …, 2023 - thelancet.com
Background Previous global analyses, with known underdiagnosis and single cause per
death attribution systems, provide only a small insight into the suspected high population …

Blood rheology: key parameters, impact on blood flow, role in sickle cell disease and effects of exercise

E Nader, S Skinner, M Romana, R Fort… - Frontiers in …, 2019 - frontiersin.org
Blood viscosity is an important determinant of local flow characteristics, which exhibits shear
thinning behavior: it decreases exponentially with increasing shear rates. Both hematocrit …

Continuous evolution of SpCas9 variants compatible with non-G PAMs

SM Miller, T Wang, PB Randolph, M Arbab… - Nature …, 2020 - nature.com
The targeting scope of Streptococcus pyogenes Cas9 (SpCas9) and its engineered variants
is largely restricted to protospacer-adjacent motif (PAM) sequences containing G bases …

[HTML][HTML] A red carpet for iron metabolism

MU Muckenthaler, S Rivella, MW Hentze, B Galy - Cell, 2017 - cell.com
200 billion red blood cells (RBCs) are produced every day, requiring more than 2× 10 15
iron atoms every second to maintain adequate erythropoiesis. These numbers translate into …

[HTML][HTML] Crizanlizumab for the prevention of pain crises in sickle cell disease

KI Ataga, A Kutlar, J Kanter, D Liles… - … England Journal of …, 2017 - Mass Medical Soc
Background The up-regulation of P-selectin in endothelial cells and platelets contributes to
the cell–cell interactions that are involved in the pathogenesis of vaso-occlusion and sickle …

Understanding sickle cell disease: causes, symptoms, and treatment options

C Elendu, DC Amaechi, CE Alakwe-Ojimba… - Medicine, 2023 - journals.lww.com
Sickle cell disease (SCD) is a hereditary blood disorder characterized by the production of
abnormal hemoglobin molecules that cause red blood cells to take on a crescent or sickle …

Global burden of sickle cell anaemia in children under five, 2010–2050: modelling based on demographics, excess mortality, and interventions

FB Piel, SI Hay, S Gupta, DJ Weatherall… - PLoS …, 2013 - journals.plos.org
Background The global burden of sickle cell anaemia (SCA) is set to rise as a consequence
of improved survival in high-prevalence low-and middle-income countries and population …

[HTML][HTML] Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates

FB Piel, AP Patil, RE Howes, OA Nyangiri, PW Gething… - The Lancet, 2013 - thelancet.com
Background Reliable estimates of populations affected by diseases are necessary to guide
efficient allocation of public health resources. Sickle haemoglobin (HbS) is the most …