Subretinal Gene Therapy for Treatment of Retinal and Choroidal Vascular Diseases

•Subretinal injections are theorized to reduce risk of adverse immune response.•Subretinal gene therapy mitigates risk of transgene insertion in unintentional tissues.•Previous trials have demonstrated safety and tolerability of subretinal gene therapy.•ABBV-RGX-314 subretinal gene therapy has been...

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Veröffentlicht in:American journal of ophthalmology 2024-12
Hauptverfasser: Khanani, Arshad M., Aziz, Aamir A., Khanani, Zoha A., Khan, Hannah, Mojumder, Ohidul, Sulahria, Humza, Khanani, Ibrahim, Khan, Huma, Gahn, Greggory M., Mishra, Kapil
Format: Artikel
Sprache:eng
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Zusammenfassung:•Subretinal injections are theorized to reduce risk of adverse immune response.•Subretinal gene therapy mitigates risk of transgene insertion in unintentional tissues.•Previous trials have demonstrated safety and tolerability of subretinal gene therapy.•ABBV-RGX-314 subretinal gene therapy has been safe and efficacious in trials to date and is being evaluated in pivotal Phase III trials.
ISSN:0002-9394
1879-1891
1879-1891
DOI:10.1016/j.ajo.2024.12.002