Subretinal Gene Delivery Using Helper-Dependent Adenoviral Vectors
Open accessAbstract
This study describes the successful delivery of helper-dependent adenoviral vectors to the mouse retina with long term and robust levels of reporter expression in the retina without apparent adverse effects. Since these vectors have a large cloning capacity, they have great potential to extend the success of gene therapy achieved using the adeno-associated viral vector.
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HdAd-encoding CEP290 delivered to CD-1 mice resulted in long-term gene expression without a decrease.
“A long-term gene expression without a decrease was reported [93].”
From Immune responses to gene editing by viral and non-viral delivery vectors used in retinal gene therapy (2022) · cites this paper as [93]
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References 32 · Cited by 13
Research works printed but not linked: 1
- Muzyczka N Berns KI Parvoviridae: the viruses and their replication 2001 Philadelphia, Pennsylvania, USA: Lippincott Williams & Wilkins
- Authors
- Wu, L., Lam, S., Cao, H., Guan, R., Duan, R., van der Kooy, D., Bremner, R., Molday, R. S., & Hu, J.
- Affiliations
- Hospital for Sick Children (CA)
- University of Toronto (CA)
- University Health Network (CA)
- University of British Columbia (CA)
- Corresponding author
- Hu, Jim
- Venue
- Cell & Bioscience
- Published
- 2011-04
- Volume
- 1
- Issue
- 1
- Pages
- 15-15
- ISSN
- 2045-3701 (ISSN-L)
- PMC
- PMC3125205
- OpenAlex
- W2121443197
- Cited by
- 13
- References
- 32
- Citations by year
- 2026: 2 · 2025: 1 · 2024: 1 · 2022: 2 · 2017: 1 · 2016: 1 · 2014: 2 · 2013: 1 · 2012: 1
- FWCI
- 0.80
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- cc-by
- Locations
- Cell & Bioscience (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- TSpace (University of Toronto) (submittedVersion)
- cIRcle (University of British Columbia) (submittedVersion, cc-by)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Open Collections
- Funders
- Canadian Institutes of Health Research
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- helper-dependent adenoviral vectors, subretinal gene delivery, retinal gene therapy, adeno-associated virus vectors, reporter expression, long-term transgene expression, mouse retina
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