Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches

• Main Authors: Alessandro Fraldi (Author), Marta Serafini (Author), Nicolina Cristina Sorrentino (Author), Bernhard Gentner (Author), Alessandro Aiuti (Author), Maria Ester Bernardo (Author)
• פורמט: ספר
• יצא לאור: BMC, 2018-11-01T00:00:00Z.
• נושאים: article
• גישה מקוונת: Connect to this object online.

Abstract Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders caused by a deficiency in lysosomal enzymes catalyzing the stepwise degradation of glycosaminoglycans (GAGs). The current therapeutic strategies of enzyme replacement therapy and allogeneic hematopoietic stem cell transp...