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 …

Sickle cell disease: combination new therapies vs. CRISPR-Cas9 potential and challenges

I Youssry, N Ayad - Annals of Hematology, 2024 - Springer
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 …

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 …

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 …

[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 …

[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 …

[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 …

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 …

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 …

[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 …