Search

Sickle cell disease (SCD) results from a sequence defect in the β‐globin chain of adult hemoglobin (HbA) leading to expression of sickle hemoglobin (HbS). It is traditionally diagnosed by cellulose‐acetate hemoglobin electrophoresis or high‐performance liquid chromatography. While clinically useful, these methods have both sensitivity and specificity limitations. We developed a novel mass spectrometry (MS) method for the rapid, sensitive and highly quantitative detection of endogenous human β‐globin and sickle hβ‐globin, as well as lentiviral‐encoded therapeutic hβAS3‐globin in cultured cells and small quantities of mouse peripheral blood. The MS methods were used to phenotype homozygous HbA (AA), heterozygous HbA–HbS (AS) and homozygous HbS (SS) Townes SCD mice and detect lentiviral vector‐encoded hβAS3‐globin in transduced mouse erythroid cell cultures and transduced human CD34+ cells after erythroid differentiation. hβAS3‐globin was also detected in peripheral blood 6 weeks post‐transplant of transduced Townes SS bone marrow cells into syngeneic Townes SS mice and persisted for over 20 weeks post‐transplant. As several genome‐editing and gene therapy approaches for severe hemoglobin disorders are currently in clinical trials, this MS method will be useful for patient assessment before treatment and during follow‐up. [ABSTRACT FROM AUTHOR]

Aim: To compare the success rate and complications of adjuvant use of mitomycin C and triamcinolone-impregnated biodegradable nasal packing (TABP) in endoscopic dacryocystorhinostomy (DCR). And to evaluate the efficacy of combining intraoperative mitomycin C and TABP for endoscopic DCR., Methods: A total of 198 eyes of 148 patients who underwent endoscopic DCR for acquired nasolacrimal duct obstruction were retrospectively analysed. The patients were randomly divided into three groups: Group A included patients treated without intraoperative mitomycin C but with TABP, Group B included patients treated without triamcinolone but with intraoperative mitomycin C and normal saline-impregnated nasal packing, and Group C included patients treated with intraoperative mitomycin C and TABP., Results: The results revealed no significant difference in the overall success rates between Groups A (86.8%) and B (89.2%; P =0.377). However, Group C (97.5%) showed a significantly higher overall success rate than Groups A and B. The incidence of granulomas was significantly lower in group C (5%) than in Groups A (20.8%) and B (15.2%; P =0.009). Other complications, such as crust, synechiae, and revision surgery, did not differ significantly among the three groups., Conclusion: The combination of intraoperative mitomycin C and TABP effectively prevents granulomas and enhances surgical success rate. Additionally, there is no statistically significant difference observed between the use of mitomycin C or TABP alone., Competing Interests: Conflicts of Interest: Kim SY, None; Chambers CB, None; Ahn JH, None., (International Journal of Ophthalmology Press.)