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Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts
Published on: December 17, 2016
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Systemic IGF-1 gene delivery by rAAV9 improves spontaneous autoimmune peripheral polyneuropathy (SAPP)
Tong Gao1, Nataliia Bogdanova1, Sameera Ghauri1
1Department of Neurology, McGovern Medical School at The University of Texas Health Science Center at Houston, Houston, TX, 77030, USA.
Scientific Reports
|April 5, 2018
Summary
Gene therapy using insulin-like growth factor 1 (IGF-1) delivered via rAAV9 shows promise for treating autoimmune neuropathies. This approach improved symptoms in a mouse model, offering hope for conditions like chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).
Area of Science:
- Neuroscience
- Immunology
- Genetics
Background:
- Spontaneous autoimmune peripheral polyneuropathy (SAPP) models chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) in B7-2 gene-deficient mice.
- SAPP is characterized by progressive, multifocal inflammation, demyelination, and axonal degeneration.
- Insulin-like growth factor 1 (IGF-1) possesses neuroprotective, regenerative, and anti-inflammatory properties.
Purpose of the Study:
- To evaluate the efficacy of IGF-1 gene therapy delivered systemically via recombinant adeno-associated virus serotype 9 (rAAV9) in the SAPP mouse model.
- To assess the therapeutic potential of IGF-1 gene therapy for immune-mediated neuropathies.
Main Methods:
- Systemic delivery of rAAV9 carrying the IGF-1 gene and a reporter gene into SAPP mice.
- Monitoring of neuropathic disease progression and surrogate IGF-1 expression.
Main Results:
- Systemic rAAV9/IGF-1 gene delivery significantly improved neuropathic disease in the SAPP model.
- Therapeutic benefits were observed when administered at both presymptomatic and symptomatic stages.
- Reporter gene confirmed surrogate expression of IGF-1.
Conclusions:
- IGF-1 gene therapy, delivered via rAAV9, is a potentially effective treatment for immune-mediated neuropathies.
- This approach offers a viable therapeutic strategy for conditions like CIDP, particularly for non-responsive patients.
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