Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2020 Dec;9(4):709-724.
doi: 10.1007/s40123-020-00287-1. Epub 2020 Aug 1.

Treatment-Emergent Adverse Events in Gene Therapy Trials for Inherited Retinal Diseases: A Narrative Review

Yan Nuzbrokh  1   2   3 , Alexis S Kassotis  1 , Sara D Ragi  1   2 , Ruben Jauregui  1   2 , Stephen H Tsang  4   5   6
Affiliations
Review

Treatment-Emergent Adverse Events in Gene Therapy Trials for Inherited Retinal Diseases: A Narrative Review

Yan Nuzbrokh et al. Ophthalmol Ther. 2020 Dec.

Abstract

Patient safety is a primary priority in the conduction of retinal gene therapy trials. An understanding of risk factors and mitigation strategies for post-procedure complications is crucial for the optimization of gene therapy clinical trial protocols. In this review, we synthesize the literature on ocular delivery methods, vector platforms, and treatment-emergent adverse effects in recent gene therapy clinical trials for inherited retinal diseases.

Keywords: Adverse events; Inherited retinal diseases; Retinal gene therapy.

PubMed Disclaimer

Conflict of interest statement

Yan Nuzbrokh, Alexis S. Kassotis, Sara D. Ragi, Ruben Jauregui and Stephen H. Tsang declare they have no conflicts of interest.

References

    1. FDA. FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss. In: https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm589467.html. Accessed July 16, 2020.
    1. Ong T, Pennesi ME, Birch DG, et al. Adeno-associated viral gene therapy for inherited retinal disease. Pharm Res. 2019;36:34. doi: 10.1007/s11095-018-2564-5. - DOI - PMC - PubMed
    1. Zhou R, Caspi RR. Ocular immune privilege. F1000 Biol Rep. 2010;2:3. doi: 10.3410/B2-3. - DOI - PMC - PubMed
    1. Han Z, Conley SM, Naash MI. AAV and compacted DNA nanoparticles for the treatment of retinal disorders: challenges and future prospects. Invest Ophthalmol Vis Sci. 2011;52:3051–3059. doi: 10.1167/iovs.10-6916. - DOI - PMC - PubMed
    1. Zufferey R, Dull T, Mandel RJ, et al. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J Virol. 1998;72:9873–9880. doi: 10.1128/JVI.72.12.9873-9880.1998. - DOI - PMC - PubMed

LinkOut - more resources