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Gene therapy for the lysosomal storage disorders.
Mario A Cabrera-Salazar1, Enrico Novelli, John A Barranger
1Department of Human Genetics, University of Pittsburgh, PA 15261, USA.
Current Opinion in Molecular Therapeutics
|September 12, 2002
Summary
Gene therapy offers a promising alternative for lysosomal storage disorders (LSDs), particularly for neurological symptoms unresponsive to current treatments like enzyme replacement therapy (ERT). This review explores gene therapy strategies for LSDs, including those for Gaucher disease Type 1.
Area of Science:
- Biochemistry
- Genetics
- Cell Biology
Background:
- Lysosomal storage disorders (LSDs) are inherited metabolic diseases with diverse clinical presentations.
- Bone marrow transplantation (BMT) and enzyme-replacement therapy (ERT) show promise but have limitations such as donor availability, high costs, and incomplete efficacy, especially for neurological manifestations.
Purpose of the Study:
- To review current and developing gene therapy strategies for lysosomal storage disorders (LSDs).
- To evaluate the potential of gene therapy as an alternative treatment for both neurological and non-neurological LSDs.
Main Methods:
- Review of preclinical in vitro and in vivo studies.
- Analysis of oncoretroviral, adeno-associated, and lentiviral vector applications in gene therapy for LSDs.
- Examination of studies providing rationale for clinical trials, such as for Gaucher disease Type 1.
Main Results:
- Preclinical studies demonstrate proof-of-concept for BMT and ERT in LSDs.
- Gene therapy using various viral vectors has shown success in preclinical models.
- Gene therapy is identified as a key alternative for addressing neuropathology in LSDs.
Conclusions:
- Gene therapy presents a viable and attractive therapeutic strategy for LSDs, especially for neurological symptoms.
- Preclinical gene therapy research supports the initiation of clinical trials for specific LSDs like Gaucher disease Type 1.
- Further development of gene therapy vectors and strategies is crucial for effective LSD treatment.