Abstract
Lysosomal storage disorders (LSDs) are a group of diseases with multisystemic features. Current treatments have limitations and gene therapy arises as a promising treatment option. Here, we discuss some of the most recent studies for gene therapy in LSD, vectors used, and outcomes. In particular, the approaches used in animal models aiming to correct the central nervous system, the eye, and the bones are highlighted. Finally, we discuss the recent reports of clinical trials using this technology for these diseases. We conclude that gene therapy for LSD has gathered a substantial amount of evidence from animal models to know its potential and limitations. First evidences from clinical trials using both adeno-associated and lentiviral vectors show that this approach is safe and efficient and therefore could provide an effective treatment for several LSD in the near future.
Highlights
SummaryHigher levels of circulating enzyme are needed to correct the heart valves
Lysosomal storage disorders (LSDs) are a group of diseases with multisystemic features
Lysosomal storage disorders (LSDs) are a group of about 60 heterogeneous diseases that result in lysosomal dysfunction, usually as a consequence of deficiency of an enzyme required for the metabolism of complex molecules
Summary
Higher levels of circulating enzyme are needed to correct the heart valves These structures are especially affected in different MPS, other LSDs such as galactosialidosis can show valve disease.[13] Mucopolysaccharidoses results from deficiency in one of the several enzymes involved in the. Both viral and nonviral vectors have been delivered either directly into the CNS or in the circulation. These vectors were very effective in correcting disease phenotype, quickly becoming one of the most promising approaches for the treatment of LSD.
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