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Gene therapy for lysosomal storage diseases: progress, challenges and future prospects
Sergey S Seregin1, Andrea Amalfitano
1Department of Microbiology and Molecular Genetics, College of Osteopathic Medicine, Michigan State University, East Lansing, MI 48824, USA.
Current Pharmaceutical Design
|July 22, 2011
Summary
Lysosomal storage diseases (LSDs) are inherited disorders caused by enzyme deficiencies. Gene therapy shows promise for treating LSDs, but requires understanding disease biology and immune responses.
Area of Science:
- Biochemistry
- Genetics
- Medicine
Background:
- Lysosomal Storage Diseases (LSDs) are inherited metabolic disorders affecting ~1 in 7,000 births.
- Caused by genetic deficiencies in lysosomal enzymes, leading to macromolecule accumulation.
- LSDs result in significant morbidity, mortality, and currently have no cure.
Purpose of the Study:
- To review current treatment options for LSDs.
- To explore the potential of gene therapy for treating LSDs.
- To highlight advances and remaining challenges in gene therapy for LSDs.
Main Methods:
- Review of existing literature on LSDs and gene therapy.
- Analysis of data from human patients and animal models.
- Description of various viral and non-viral gene transfer platforms.
Main Results:
- LSDs exhibit variable symptoms based on gene defects and affected cells.
- Significant research data exists for LSDs in humans and animal models.
- Gene therapy approaches are being developed and optimized for LSD treatment.
Conclusions:
- LSDs may be highly amenable to gene therapy interventions.
- Overcoming limitations requires a deep understanding of LSD biology.
- Host immune responses to gene transfer must be considered for successful therapy.
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