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Sickle cell disease (SCD) is a monogenetic disorder due to a single base-pair point mutation in the β-globin gene resulting in the substitution of the amino acid valine for glutamic acid in the β-globin chain. Phenotypic variation in the clinical presentation and disease outcome is a characteristic feature of the disorder.
- Gene Therapy for Sickle Cell Disease: An Update - PMC
Allogenic hematopietic stem cell transplantation is the only...
- Gene Therapy for Sickle Cell Disease: An Update - PMC
15 Μαρ 2024 · Gene therapy is potentially a curative treatment for sickle cell disease. Early findings show lower occurrence of vaso-occlusive crisis and reduced haemolysis, although longer follow-up is still necessary to evaluate adverse effects.
Anti-sickling protein coding gene addition, fetal globin induction via knocking-down/ silencing of repressors of γ-globin gene, and sickle mutation correction with genome engineering tools, particularly CRISPR/Cas9, are the main genetic approaches for sickle cell disease.
Allogenic hematopietic stem cell transplantation is the only known cure for the disease with high success rates but the limited availability of matched sibling donors and the high risk of transplantation-related side effects force the scientific community to envision additional therapies.
16 Νοε 2018 · The objectives of this review are: ‐ to determine whether gene therapy can improve survival and prevent symptoms and complications associated with sickle cell disease; ‐ to examine the risks of gene therapy against the potential long‐term gain for people with sickle cell disease.
1 Ιουλ 2018 · In this review, general gene therapy approaches for SCD including gene addition and genome editing technologies for decreasing SCD symptoms by enhancing HbF or correcting the mutation in the β-globin sequence are outlined.
19 Ιουλ 2024 · Despite their enhanced effectiveness, approved drugs exhibited drawbacks like poor bioavailability, limited patient tolerance, and unwanted side effects.
