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Gene therapy progress and prospects: gene therapy of lysosomal storage disorders
Abstract:
Despite disappointments with early clinical studies, there is continued interest in the development of gene therapy for the group of metabolic diseases referred to as lysosomal storage disorders (LSDs). The LSDs are monogenic and several small and large, representative animal models of the human diseases are available. Further, the successful reconstitution of only low and unregulated tissue levels of the affected lysosomal enzymes are expected to be sufficient to correct the disease at least in the case of some of the LSDs. For these reasons, they are perceived as good models for the evaluation of different gene delivery vectors and of different strategies for treating chronic genetic diseases by gene transfer. In this review, we will highlight the progress that has been made over the past 2 years in preclinical research for this group of disorders and speculate on future prospects.
Insights
Gene therapy shows promise for lysosomal storage disorders (LSDs), a group of genetic diseases. Preclinical research advances in gene transfer strategies and delivery vectors offer hope for treating these rare metabolic conditions.
Area of Science:
- Biochemistry
- Genetics
- Molecular Biology
Background:
- Lysosomal storage disorders (LSDs) are a group of monogenic metabolic diseases.
- Despite early setbacks, gene therapy is a continued area of interest for LSDs.
- Animal models for LSDs are available for research.
Purpose of the Study:
- To review recent progress in preclinical gene therapy for LSDs.
- To evaluate gene delivery vectors and gene transfer strategies for LSDs.
- To discuss future prospects for gene therapy in treating LSDs.
Main Methods:
- Review of preclinical research over the past two years.
- Analysis of gene delivery vectors.
- Evaluation of gene transfer strategies.
Main Results:
- Progress has been made in preclinical gene therapy research for LSDs.
- Lysosomal enzyme levels may be sufficient for disease correction in some LSDs.
- LSD models are valuable for evaluating gene therapy approaches.
Conclusions:
- Gene therapy holds potential for treating LSDs.
- Further research is needed to optimize gene transfer strategies and vectors.
- LSDs serve as important models for developing chronic genetic disease treatments.