Journal
FRONTIERS IN MOLECULAR NEUROSCIENCE
Volume 7, Issue -, Pages -Publisher
FRONTIERS MEDIA SA
DOI: 10.3389/fnmol.2014.00089
Keywords
adeno-associated virus; systemic delivery; perinatal; gene therapy; neurodegenerative disease; mouse models; non-human primates; metabolic disease
Categories
Funding
- UK Medical Research Council [G1000709]
- Gauchers Association
- Niemann-Pick Disease Group
- Niemann-Pick Research Foundation
- US Neurodegeneration with Brain iron Accumulation Disorders Association
- Action Medical Research [2137] Funding Source: researchfish
- Great Ormond Street Hospital Childrens Charity [V1284] Funding Source: researchfish
- Medical Research Council [G1000709, MR/K02342X/1] Funding Source: researchfish
- MRC [G1000709, MR/K02342X/1] Funding Source: UKRI
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Neurodegenerative monogenic diseases often affect tissues and organs beyond the nervous system. An effective treatment would require a systemic approach. The intravenous administration of novel therapies is ideal but is hampered by the inability of such drugs to cross the blood brain barrier (BBB) and precludes efficacy in the central nervous system. A number of these early lethal intractable diseases also present devastating irreversible pathology at birth or soon after. Therefore, any therapy would ideally be administered during the perinatal period to prevent, stop, or ameliorate disease progression. The concept of perinatal gene therapy has moved a step further toward being a feasible approach to treating such disorders. This has primarily been driven by the recent discoveries that particular serotypes of adeno-associated virus (AAV) gene delivery vectors have the ability to cross the BBB following intravenous administration. Furthermore, safety has been demonstrated after perinatal administration mice and non-human primates. This review focuses on the progress made in using AAV to achieve systemic transduction and what this means for developing perinatal gene therapy for early lethal neurodegenerative diseases.
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