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Gene therapy for lysosomal storage disorders.
Yiannis A Ioannou1, Annette Enriquez, Compton Benjamin
1Departments of Human Genetics, Gene Therapy & Molecular Medicine, The Mount Sinai School of Medicine, New York, NY, USA, NY 10029, USA. Yiannis.Ioannou@mssm.edu
Expert Opinion on Biological Therapy
|July 26, 2003
Summary
Gene therapy offers promising treatment for lysosomal storage disorders (LSDs), especially those affecting the brain. While effective in vivo and ex vivo, challenges remain before human trials can begin.
Area of Science:
- Genetics
- Molecular Biology
- Biochemistry
Background:
- Lysosomal storage disorders (LSDs) are a significant group of inherited metabolic diseases.
- Current therapies are limited for many LSDs, particularly those with neurological involvement.
- Enzyme replacement therapies show success in non-neuropathic LSDs, driving gene therapy research.
Purpose of the Study:
- To review the progress and potential of gene therapy for treating lysosomal storage disorders.
- To highlight gene therapy as a key therapeutic option for LSDs with neuropathology.
- To identify challenges hindering the clinical application of gene therapy for LSDs.
Main Methods:
- Review of existing studies on gene therapy for LSDs.
- Analysis of in vivo and ex vivo gene therapy approaches.
- Evaluation of various vector systems used in gene therapy for LSDs.
Main Results:
- Gene therapy, utilizing diverse vectors, demonstrates significant promise for LSD treatment.
- Both in vivo and ex vivo strategies show potential for therapeutic success.
- Enzyme replacement therapy success in non-neuropathic LSDs provides a foundation for gene therapy development.
Conclusions:
- Gene therapy is a leading therapeutic strategy for LSDs, especially those impacting the central nervous system.
- Despite promising results, several critical issues must be addressed for safe and effective human gene therapy trials.
- Further research is essential to overcome existing hurdles in LSD gene therapy.