Breaking genetic shackles: The advance of base editing in genetic disorder treatment
F Xu, C Zheng, W Xu, S Zhang, S Liu… - Frontiers in …, 2024 - frontiersin.org
The rapid evolution of gene editing technology has markedly improved the outlook for
treating genetic diseases. Base editing, recognized as an exceptionally precise genetic …
treating genetic diseases. Base editing, recognized as an exceptionally precise genetic …
Sickle cell disease: combination new therapies vs. CRISPR-Cas9 potential and challenges
In 2022, sickle cell disease (SCD) continues to affect the lives of millions of people, being
one of the most frequently inherited blood disorders worldwide. Recently, several new …
one of the most frequently inherited blood disorders worldwide. Recently, several new …
Excellent outcome of stem cell transplantation for sickle cell disease
T Vallée, I Schmid, L Gloning, M Bacova, J Ahrens… - Annals of …, 2023 - Springer
Many sickle cell disease (SCD) patients lack matched family donors (MFD) or matched
unrelated donors (MUD), implying haploidentical donors (MMFD) as a logical donor choice …
unrelated donors (MUD), implying haploidentical donors (MMFD) as a logical donor choice …
Opportunity for pharmacogenetics testing in patients with sickle cell anemia
KA Gallaway, C Sakon, J Ongeri, KS Patel… - …, 2022 - Taylor & Francis
Background: Patients with sickle cell disease (SCD) are exposed to numerous drugs over
their lifespan, and many of these drugs have Clinical Pharmacogenetics Implementation …
their lifespan, and many of these drugs have Clinical Pharmacogenetics Implementation …
[HTML][HTML] Review on Hydroxyurea Usage in Young Children with Sickle Cell Disease: Examining Hemoglobin Induction, Potential Benefits, Responses, Safety, and …
MC Mkwambe, Y Deng, D Zhao - International Journal of Clinical Medicine, 2024 - scirp.org
Sickle cell disease (SCD) is a prevalent condition, particularly in the countries of sub-
Saharan Africa, where the presence of specific genes associated with Malaria contributes to …
Saharan Africa, where the presence of specific genes associated with Malaria contributes to …
[HTML][HTML] Hemoglobin Variants as Targets for Stabilizing Drugs
M Žoldáková, M Novotný, KP Khakurel, G Žoldák - Molecules, 2025 - pmc.ncbi.nlm.nih.gov
Hemoglobin is an oxygen-transport protein in red blood cells that interacts with multiple
ligands, eg, oxygen, carbon dioxide, carbon monoxide, and nitric oxide. Genetic variations in …
ligands, eg, oxygen, carbon dioxide, carbon monoxide, and nitric oxide. Genetic variations in …
[HTML][HTML] Identification of proinflammatory pathways and promising bioactive polyphenols for the treatment of sickle cell anemia by in silico study and network …
LR Maturana-Pérez, J Márquez-Lázaro… - Informatics in Medicine …, 2024 - Elsevier
Sickle cell anemia (SCA) is an autosomal recessive Mendelian trait characterized by
symptoms that include acute and chronic pain, chest syndrome, pulmonary hypertension …
symptoms that include acute and chronic pain, chest syndrome, pulmonary hypertension …
Effect of nutritional supplementation on bone mineral density in children with sickle cell disease: protocol for an open-label, randomised controlled clinical trial
M Condé, E Lespessailles, M Wanneveich… - BMJ open, 2024 - bmjopen.bmj.com
Introduction Children with sickle cell disease show a significant decrease in bone mineral
density, an increase in resting energy expenditure of more than 15%, a decrease in fat and …
density, an increase in resting energy expenditure of more than 15%, a decrease in fat and …
Colchicine reduces inflammation in a humanized transgenic murine model of sickle cell disease
RT Fouda, HM Cherukury, SB Kiven… - …, 2023 - pmc.ncbi.nlm.nih.gov
Sickle cell diseases (SCD) are caused by a mutation in the β-globin gene that causes
deoxygenated hemoglobin to polymerize, resulting in rigid, fragile, sickle-shaped red blood …
deoxygenated hemoglobin to polymerize, resulting in rigid, fragile, sickle-shaped red blood …
[HTML][HTML] Protocol: Effect of nutritional supplementation on bone mineral density in children with sickle cell disease: protocol for an open-label, randomised controlled …
M Condé, E Lespessailles, M Wanneveich… - BMJ Open, 2024 - ncbi.nlm.nih.gov
Introduction Children with sickle cell disease show a significant decrease in bone mineral
density, an increase in resting energy expenditure of more than 15%, a decrease in fat and …
density, an increase in resting energy expenditure of more than 15%, a decrease in fat and …