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β-血红蛋白病基因组编辑治疗的研究进展 被引量:4
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作者 刘佳伟 洪涛 +3 位作者 秦鑫 梁英民 张萍 徐湘民 《遗传》 CAS CSCD 北大核心 2018年第2期95-103,共9页
β-血红蛋白病(β-hemoglobinopathies)是严重危害人类健康的6种常见疾病之一,尽管其遗传分子机制已被阐释清楚,但目前除异基因骨髓移植外尚还缺乏根治性的治疗策略。近年来,基因组编辑技术的迅速发展及其在人造血干祖细胞中的应用为β... β-血红蛋白病(β-hemoglobinopathies)是严重危害人类健康的6种常见疾病之一,尽管其遗传分子机制已被阐释清楚,但目前除异基因骨髓移植外尚还缺乏根治性的治疗策略。近年来,基因组编辑技术的迅速发展及其在人造血干祖细胞中的应用为β-血红蛋白病的治疗开辟了新的方向。针对血红蛋白的基因突变,可利用同源重组介导的细胞内源性DNA损伤修复途径直接修复遗传缺陷,也可以利用非同源末端连接机制沉默抑制胎儿血红蛋白表达的分子,重新激活胎儿血红蛋白的表达以缓解β-血红蛋白病人的临床症状。本文从β-血红蛋白病的基因组编辑策略及尝试、临床转化平台的要素分析等方面对该病的基因组编辑治疗的研究进展展开综述,以期为β-血红蛋白病新型治愈方案的研究以及基因组编辑技术的临床转化提供参考。 展开更多
关键词 β-血红蛋白病 基因组编辑 镰状红细胞贫血 Β-地中海贫血 Cas9
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Atomic modulation of Fe‐Co pentlandite coupled with nitrogen‐doped carbon sphere for boosting oxygen catalysis
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作者 Si‐Jie Li Yong Xie +5 位作者 Bi‐Lin Lai yingmin liang Kang Xiao Ting Ouyang Nan Li Zhao‐Qing Liu 《Chinese Journal of Catalysis》 SCIE EI CAS CSCD 2022年第6期1502-1510,共9页
Reversible oxygen reaction plays a crucial role in rechargeable battery systems,but it is limited by the slow reaction kinetics.Herein,the ionic modulation of cobalt pentlandite coupled with nitrogen‐doped bowl‐like... Reversible oxygen reaction plays a crucial role in rechargeable battery systems,but it is limited by the slow reaction kinetics.Herein,the ionic modulation of cobalt pentlandite coupled with nitrogen‐doped bowl‐like hollow carbon sphere is well designed on octahedral and tetrahedral sites.The robust FexCo9−xS8‐NHCS‐V with iron replacing at the octahedron possesses prolonged metal sulfur bond and exhibits excellent bifunctional electrocatalytic performance towards oxygen reduction reaction(ORR,E_(1/2)=0.80 V vs.RHE)and excellent oxygen evolution reaction(OER,E_(j=10)=1.53 V vs.RHE)in 0.1 mol/L KOH.Accordingly,a rechargeable Zn‐air battery of Fe_(x)Co_(9−x)S_(8)‐NHCS‐V cathode endows high energy efficiency(102 mW cm^(−2)),and a microbial fuel cell achieves a high‐power density(791±42 mW m^(−2)),outperforming the benchmark Pt/C catalyst. 展开更多
关键词 Oxygen electrocatalysis Transition metal sulfide Nitrogen‐doped carbon Pentlandite structure Zinc‐air battery
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老反应、新认识:不容小觑的乙硼烷与氨的反应
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作者 李慧珍 李运辉 +3 位作者 庄惠芬 陈丽婷 梁瑛敏 易思远 《大学化学》 CAS 2021年第8期43-55,共13页
看似简单实际却很复杂的乙硼烷(B_(2)H_(6),diborane)与氨(NH_(3))的反应已经困扰了硼化学家数十年。该反应在不同实验条件下可生成硼氢化二氢二氨合硼(III)([NH_(3)BH_(2)NH_(3)]BH_(4),diammoniate of diborane,DADB),氨硼烷(NH_(3)&#... 看似简单实际却很复杂的乙硼烷(B_(2)H_(6),diborane)与氨(NH_(3))的反应已经困扰了硼化学家数十年。该反应在不同实验条件下可生成硼氢化二氢二氨合硼(III)([NH_(3)BH_(2)NH_(3)]BH_(4),diammoniate of diborane,DADB),氨硼烷(NH_(3)·BH_(3),ammonia borane,AB),氨基乙硼烷(NH_(2)B_(2)H_(5),aminodiborane,ADB)等化合物。从1923年起,化学家们对影响乙硼烷与路易斯碱反应的因素,如立体效应、诱导效应、溶剂效应、介电常数等进行研究,但一直无法根据上述影响因素准确预测产物。基于乙硼烷在THF中以THF·BH_(3)存在、路易斯碱与乙硼烷或THF·BH_(3)反应产物相同等实验事实,化学家们巧妙利用NH_(3)与THF·BH_(3)的反应,在温和的条件下研究了DADB、AB的生成机理,发现双氢键是控制反应的重要因素。利用AB与THF·BH_(3)的反应,实验和理论计算结果进一步揭示了基于双氢键的ADB的复杂形成机理。 展开更多
关键词 硼氮氢化合物 乙硼烷 硼氢化二氢二氨合硼(III) 氨硼烷 氨基乙硼烷 双氢键 路易斯碱
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Reducing systemic absorption and macrophages clearance of genistein by lipid-coated nanocrystals for pulmonary delivery 被引量:1
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作者 Yuan He Chang Liu +5 位作者 Run Han yingmin liang Judith Choi Wo Mak Yinghao Zhu Haifeng Li Ying Zheng 《Chinese Chemical Letters》 SCIE CAS CSCD 2023年第1期245-250,共6页
Pulmonary delivery is an effective drug delivery strategy for the treatment of local respiratory diseases.However,the rapid systemic absorption through the lung due to the thin barrier and persistent lung clearances i... Pulmonary delivery is an effective drug delivery strategy for the treatment of local respiratory diseases.However,the rapid systemic absorption through the lung due to the thin barrier and persistent lung clearances influence the drug retention in the lung.In this study,we designed a lipid-coated genistein nanocrystals(Lipo-NCs)formulation to achieve enhanced efficiency of local pulmonary delivery.The LipoNCs were fabricated by modifying genistein nanocrystals(NCs)with phospholipid membrane through thin film hydration following the homogenization method.The prepared Lipo-NCs exhibited a decreased drug release rate compared with the naked NCs.Our results demonstrated that intracellular uptake and transcellular transport of NCs by the Calu-3 epithelial layer were reduced after lipid coating.Furthermore,the macrophages clearance was also impeded by this Lipo-NCs formulation.In vivo lung retention and distribution revealed that more genistein was retained in the lung after intratracheal administration of Lipo-NCs.The pharmacokinetic study displayed that the AUC((0-t))values of Lipo-NCs were 1.59-fold lesser than those of the NCs group,indicating a reduced systemic absorption.In conclusion,this research indicated that Lipo-NCs could be a suitable formulation for reducing systemic absorption and macrophages clearance,and thus enhancing drug concentration in lung by pulmonary delivery. 展开更多
关键词 Pulmonary delivery Lipid-coated nanocrystals Cell uptake Transport Systemic absorption Macrophage clearances
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Clinical risk score for invasive fungal diseases in patients with hematological malignancies undergoing chemotherapy:China Assessment of Antifungal Therapy in Hematological Diseases (CAESAR) study 被引量:12
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作者 Ling Wang Ying Wang +16 位作者 Jiong Hu Yuqian Sun He Huang Jing Chen Jianyong Li Jun Ma Juan Li yingmin liang Jianmin Wang Yan Li Kang Yu Jianda Hu Jie Jin Chun Wang Depei Wu Yang Xiao Xiaojun Huang 《Frontiers of Medicine》 SCIE CAS CSCD 2019年第3期365-377,共13页
Invasive fungal disease (IFD) is a major infectious complication in patients with hematological malignancies.In this study,we examined 4889 courses of chemotherapy in patients with hematological diseases to establish ... Invasive fungal disease (IFD) is a major infectious complication in patients with hematological malignancies.In this study,we examined 4889 courses of chemotherapy in patients with hematological diseases to establish a training dataset (n=3500) by simple random sampling to develop a weighted risk score for proven or probable IFD through multivariate regression,which included the following variables: male patients,induction chemotherapy for newly diagnosed or relapsed disease,neutropenia,neutropenia longer than 10 days,hypoalbuminemia,central-venous catheter,and history of IFD.The patients were classified into three groups,which had low (0-10,~1.2%),intermediate (11-15,6.4%),and high risk (> 15,17.5%) of IFD.In the validation set (n=1389),the IFD incidences of the groups were ~1.4%,5.0%,and 21.4%.In addition,we demonstrated that antifungal prophylaxis offered no benefits in low-risk patients,whereas benefits were documented in intermediate (2.1% vs.6.6%,P=0.007) and high-risk patients (8.4% vs.23.3%,P=0.007).To make the risk score applicable for clinical settings,a pre-chemo risk score that deleted all unpredictable factors before chemotherapy was established,and it confirmed that anti-fungal prophylaxis was beneficial in patients with intermediate and high risk of IFD.In conclusion,an objective,weighted risk score for IFD was developed,and it may be useful in guiding antifungal prophylaxis. 展开更多
关键词 invasive FUNGAL diseases HEMATOLOGICAL MALIGNANCIES CHEMOTHERAPY risk score PROPHYLAXIS
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