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Updated: Jul 6, 2026
10:33
Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes
Published on: July 23, 2016
Central nervous system therapy for lysosomal storage disorders
Gregory M Enns1, Stephen L Huhn
1Division of Medical Genetics, Department of Pediatrics, Lucile Packard Children's Hospital, Stanford University, Stanford, USA. greg.enns@stanford.edu
Neurosurgical Focus
|March 18, 2008
Summary
Lysosomal storage disorders cause progressive neurodegeneration. Novel therapies targeting the blood-brain barrier offer hope for treating these devastating central nervous system conditions in children.
Area of Science:
- Neurology
- Genetics
- Biochemistry
Background:
- Lysosomal storage disorders (LSDs) frequently cause progressive central nervous system (CNS) impairment.
- Neurodegeneration, following initial normal development, is a hallmark of many LSDs.
- The blood-brain barrier (BBB) poses a significant challenge for treating CNS diseases.
Purpose of the Study:
- To review current and emerging therapeutic strategies for LSDs affecting the CNS.
- To highlight advancements in overcoming the BBB for neurological treatments.
Main Methods:
- Review of current literature on LSDs and therapeutic interventions.
- Analysis of novel approaches including enzyme replacement, small-molecule, gene, and cell-based therapies.
- Discussion of the challenges and potential of BBB penetration for LSD treatment.
Main Results:
- Significant progress has been made in developing therapies for LSDs.
- Enzyme replacement, small-molecule, gene, and cell-based therapies show promise.
- Overcoming the BBB remains a critical area for therapeutic development.
Conclusions:
- Emerging therapies offer new hope for children with LSDs.
- Targeting the CNS effectively is crucial for improving outcomes in LSDs.
- Continued research into BBB-penetrating therapies is essential.
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