Mucopolysaccharidoses typeⅢB is a rare genetic disorder caused by mutations in the gene that encodes for N-acetyl-alpha-glucosaminidase.This results in the aggregation of heparan sulfate polysaccharides within cell l...Mucopolysaccharidoses typeⅢB is a rare genetic disorder caused by mutations in the gene that encodes for N-acetyl-alpha-glucosaminidase.This results in the aggregation of heparan sulfate polysaccharides within cell lysosomes that leads to progressive and severe debilitating neurological dysfunction.Current treatment options are expensive,limited,and presently there are no approved cures for mucopolysaccharidoses typeⅢB.Adeno-associated virus gene therapy has significantly advanced the field forward,allowing researchers to successfully design,enhance,and improve potential cures.Our group recently published an effective treatment using a codon-optimized triple mutant adeno-associated virus 8 vector that restores N-acetyl-alpha-glucosaminidase levels,auditory function,and lifespan in the murine model for mucopolysaccharidoses typeⅢB to that seen in healthy mice.Here,we review the current state of the field in relation to the capsid landscape,adeno-associated virus gene therapy and its successes and challenges in the clinic,and how novel adenoassociated virus capsid designs have evolved research in the mucopolysaccharidoses typeⅢB field.展开更多
Two new triterpenoid saponins, named isoescins IIIa (1) and IIIb (2) were isolated from the seeds of Aesculus chinensis and identified by spectroscopic analysis and chemical hydrolysis. Their structures were establish...Two new triterpenoid saponins, named isoescins IIIa (1) and IIIb (2) were isolated from the seeds of Aesculus chinensis and identified by spectroscopic analysis and chemical hydrolysis. Their structures were established as 21 beta-tigloyl-28-acetylbarringtogenol C-3 beta-O-[beta D-galactopyranosyl( 1-->2)] [beta-D-glucopyranosyl (1-->])1-beta-D-glucopyranosiduronic acid (1) and 21 beta-angeloyl-28-acetylbarringtogenol C-3 beta-O-[beta-D;galactopyranosyl (1-->2)] [beta-D-glucopyranosyl(1 -->4)]-beta-D-glucopyranosiduronic acid (2), which are geometrically isomeric.展开更多
目的:观察督脉灸治疗慢性盆腔疼痛综合征寒凝血瘀证的临床疗效。方法:选择80例慢性盆腔疼痛综合征患者,辨证为寒凝血瘀证,随机数字表法分为治疗组和对照组。治疗组采用督脉灸治疗,对照组口服少腹逐瘀颗粒,每次1袋,每天3次。比较两组患...目的:观察督脉灸治疗慢性盆腔疼痛综合征寒凝血瘀证的临床疗效。方法:选择80例慢性盆腔疼痛综合征患者,辨证为寒凝血瘀证,随机数字表法分为治疗组和对照组。治疗组采用督脉灸治疗,对照组口服少腹逐瘀颗粒,每次1袋,每天3次。比较两组患者治疗前后中医证候评分、美国国立卫生研究院慢性前列腺炎症状指数评分(National Institutes of health chronic prostatitis symptom index,NIH-CPSI)、卵磷脂小体数、焦虑及抑郁自评量表评分等指标的变化,并评定两组疗效。结果:①治疗组有效率为90.0%(36/40),对照组为72.5%(29/40),治疗组优于对照组(P<0.05)。②治疗后两组中医证候积分均显著降低(均P<0.01),且治疗组优于对照组(8.56±3.17 vs 11.09±3.49,P<0.01)。③治疗后两组卵磷脂小体均明显增加(均P<0.01),且治疗组优于对照组(32.58±6.07 vs 28.45±7.32,P<0.05)。④治疗后两组NIH-CPSI总评分及疼痛、焦虑、抑郁评分均明显改善(均P<0.01),且治疗组均优于对照组(P<0.01,P<0.05)。结论:督脉灸治疗慢性盆腔疼痛综合征寒凝血瘀证临床疗效满意。展开更多
文摘Mucopolysaccharidoses typeⅢB is a rare genetic disorder caused by mutations in the gene that encodes for N-acetyl-alpha-glucosaminidase.This results in the aggregation of heparan sulfate polysaccharides within cell lysosomes that leads to progressive and severe debilitating neurological dysfunction.Current treatment options are expensive,limited,and presently there are no approved cures for mucopolysaccharidoses typeⅢB.Adeno-associated virus gene therapy has significantly advanced the field forward,allowing researchers to successfully design,enhance,and improve potential cures.Our group recently published an effective treatment using a codon-optimized triple mutant adeno-associated virus 8 vector that restores N-acetyl-alpha-glucosaminidase levels,auditory function,and lifespan in the murine model for mucopolysaccharidoses typeⅢB to that seen in healthy mice.Here,we review the current state of the field in relation to the capsid landscape,adeno-associated virus gene therapy and its successes and challenges in the clinic,and how novel adenoassociated virus capsid designs have evolved research in the mucopolysaccharidoses typeⅢB field.
文摘Two new triterpenoid saponins, named isoescins IIIa (1) and IIIb (2) were isolated from the seeds of Aesculus chinensis and identified by spectroscopic analysis and chemical hydrolysis. Their structures were established as 21 beta-tigloyl-28-acetylbarringtogenol C-3 beta-O-[beta D-galactopyranosyl( 1-->2)] [beta-D-glucopyranosyl (1-->])1-beta-D-glucopyranosiduronic acid (1) and 21 beta-angeloyl-28-acetylbarringtogenol C-3 beta-O-[beta-D;galactopyranosyl (1-->2)] [beta-D-glucopyranosyl(1 -->4)]-beta-D-glucopyranosiduronic acid (2), which are geometrically isomeric.
文摘目的:观察督脉灸治疗慢性盆腔疼痛综合征寒凝血瘀证的临床疗效。方法:选择80例慢性盆腔疼痛综合征患者,辨证为寒凝血瘀证,随机数字表法分为治疗组和对照组。治疗组采用督脉灸治疗,对照组口服少腹逐瘀颗粒,每次1袋,每天3次。比较两组患者治疗前后中医证候评分、美国国立卫生研究院慢性前列腺炎症状指数评分(National Institutes of health chronic prostatitis symptom index,NIH-CPSI)、卵磷脂小体数、焦虑及抑郁自评量表评分等指标的变化,并评定两组疗效。结果:①治疗组有效率为90.0%(36/40),对照组为72.5%(29/40),治疗组优于对照组(P<0.05)。②治疗后两组中医证候积分均显著降低(均P<0.01),且治疗组优于对照组(8.56±3.17 vs 11.09±3.49,P<0.01)。③治疗后两组卵磷脂小体均明显增加(均P<0.01),且治疗组优于对照组(32.58±6.07 vs 28.45±7.32,P<0.05)。④治疗后两组NIH-CPSI总评分及疼痛、焦虑、抑郁评分均明显改善(均P<0.01),且治疗组均优于对照组(P<0.01,P<0.05)。结论:督脉灸治疗慢性盆腔疼痛综合征寒凝血瘀证临床疗效满意。
