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Nonetheless, the aforementioned animal models, although extremely useful in investigating specific molecular mechanisms in the pathogenesis of ulcerative colitis, do not truly recapitulate disease as observed in patients, as IBD is a multifactorial disease and most clearly is not caused by a single mutation or defect in cellular or molecular pathways
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They have delved deeply into the mechanism of ferroptosis in corneal injuries and revealed the laws of the occurrence and development of the disease at the cellular and molecular levels, providing crucial clues for the development of targeted treatment strategies
Other approaches to anti-sickling gene therapy in erythroid-specific lentiviral vectors include utilizing a -globin gene with three specific point mutations that confer anti-sickling properties (ClinicalTrials.gov Identifier: NCT02247843) or the introduction of a -globin coding sequence in a -globin gene to increase HbF levels and decrease HbS (ClinicalTrials.gov Identifier: NCT02186418) ( (b) Hb F induction: The well-established efficacy of increasing HbF has motivated both pharmacological and genetic approaches to HbF induction
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