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
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.
