Self-complementary AAV vector therapy for treating corneal cloudiness of mucopolysaccharidosis type VII (MPS VII)

To design a novel efficacious scAAV-Gusb viral vector for treating Mucopolysaccharidosis Type VII (MPS VII) caused by a mutation in the β-Glu gene (Gusb allele). β-Glu expression of single-stranded AAV-Gusb (ssAAV-Gusb) and self-complementary AAV (scAAV-Gusb) vectors are tested with cultured murine...

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Veröffentlicht in:	The ocular surface 2024-04, Vol.32, p.39-47
Hauptverfasser:	Venkatakrishnan, Jhuwala, Yuan, Yong, Zhang, Jianhua, Yu, Yang, Hu, Yueh-Chiang, Kao, Winston W-Y
Format:	Artikel
Sprache:	eng
Schlagworte:	Adeno-associated vectors Animals Cells, Cultured Cornea - pathology Corneal Opacity - therapy Dependovirus - genetics Disease Models, Animal Fibroblasts Gene therapy Genetic Therapy - methods Genetic Vectors Mice Mice, Inbred C57BL Microscopy, Confocal Mucopolysaccharidosis type VII Mucopolysaccharidosis VII - genetics Mucopolysaccharidosis VII - therapy Self-complimentary AAV vectors Sly syndrome
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