Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases

被引：18

作者：

Tomanin, Rosella

Zanetti, Alessandra

Zaccariotto, Eva

D'Avanzo, Francesca

Bellettato, Cinzia M.

Scarpa, Maurizio ^{[1
]}

机构：

[1] Univ Padua, Dept Pediat, Gene Therapy Lab, I-35128 Padua, Italy

来源：

ACTA PAEDIATRICA | 2012年 / 101卷 / 07期

关键词：

Gene therapy; Lysosomal storage disorders; Mendelian diseases; CENTRAL-NERVOUS-SYSTEM; BLOOD-BRAIN-BARRIER; NEURONAL CEROID-LIPOFUSCINOSIS; ACID ALPHA-GLUCOSIDASE; LONG-TERM EXPRESSION; METACHROMATIC LEUKODYSTROPHY; IN-VIVO; MOUSE MODEL; TRANSGENE EXPRESSION; BETA-GLUCURONIDASE;

D O I：

10.1111/j.1651-2227.2012.02674.x

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

This review describes the different gene therapy technologies applied to approach lysosomal storage disorders, monogenic conditions, with known genetic and biochemical defects, for many of which animal models are available. Both viral and nonviral procedures are described, underlying the specific needs that the treatment of genetic disorders requires. Conclusions: Lysosomal storage disorders represent a good model of study of gene therapeutic procedures that are, or could be, relevant to the treatment of several other mendelian diseases.

引用

页码：692 / 701

页数：10

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