Correction: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models

Vasireddy, Vidyullatha; Mills, Jason A.; Gaddameedi, Rajashekhar; Basner-Tschakarjan, Etiena; Köhnke, Monika; Black, Aaron; Alexandrov, Krill; Zhou, Shangzhen; Maguire, Albert M.; Chung, Daniel C.; Mac, Helen; Sullivan, Lisa; Gadue, Paul; Bennicelli, Jeannette L.; French, Deborah L.; Bennett, Jean

doi:10.1371/journal.pone.0129982

Cited by 2 publications

(1 citation statement)

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“… 44 There are some challenges for gene therapy in choroideremia, including insufficient resemblance of animal models to functional and morphological manifestations of the disease, and uncertainty of which retinal layer is affected most. 45 , 46 Gene therapy outcomes for choroideremia have been less successful than RPE65 -related Leber congenital amaurosis (LCA). 47 There are currently nine clinical trials registered on ClinicalTrials.gov evaluating interventional gene therapies for these patients – all of which use AAV vectors.…”

Section: Applications Of Gene Therapy In Different Ocular Diseasesmentioning

confidence: 99%

Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses

Ghoraba¹,

Akhavanrezayat²,

Karaca³

et al. 2022

OPTH

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Gene therapy has emerged as a research topic of choice in recent years. The eye in particular is one of few organs of the body for which gene therapy has received Food and Drug Administration approval, and it remains a field of great interest for gene therapy development. However, its associated immune and inflammatory reactions may render the treatment ineffective or harmful, which are of particular concern for the eyes due to their susceptibility to inflammation. The severity of immune and inflammatory reactions depends on the choice of vector and its route of administration. Furthermore, most preclinical and clinical studies have shown that the dose of vectors is correlated with the degree of humoral response and ocular inflammation. The route of administration directly impacts the degree of immune and inflammatory reaction. Subretinal delivery produces a weaker humoral response than the intravitreal route. However, some studies have demonstrated that the subretinal delivery induces a stronger inflammatory reaction. On the other hand, several instances of vision loss due to severe late onset intraocular inflammation were reported in a clinical trial involving intravitreal delivery of viral vectors. When compared with the intravitreal route, suprachoroidal gene delivery has been shown to produce weaker humoral response. However, unlike the subretinal space, the suprachoroidal space is not known to have immune privilege status. Inflammatory reactions following ocular gene therapy are typically mild and most clinical and preclinical studies have shown that they can be controlled with topical, local or systemic steroids. However, severe inflammatory responses may occur and require aggressive management to avoid permanent vision loss. Further investigations are required to elucidate and expand our knowledge of inflammatory reactions, and their optimal management, following ocular gene therapy.

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Section: Applications Of Gene Therapy In Different Ocular Diseasesmentioning

confidence: 99%

Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses

Ghoraba¹,

Akhavanrezayat²,

Karaca³

et al. 2022

OPTH

View full text Add to dashboard Cite

show abstract

Gene therapy in inherited retinal degenerative diseases, a review

Takahashi

Takiuti

Jauregui

et al. 2018

Ophthalmic Genetics

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Hereditary diseases of the retina represent a group of diseases with several heterogeneous mutations that have the common end result of progressive photoreceptor death leading to blindness. Retinal degenerations encompass multifactorial diseases such as age-related macular degeneration, Leber congenital amaurosis, Stargardt disease, and retinitis pigmentosa. Although there is currently no cure for degenerative retinal diseases, ophthalmology has been at the forefront of the development of gene therapy, which offers hope for the treatment of these conditions. This article will explore an overview of the clinical trials of gene supplementation therapy for retinal diseases that are underway or planned for the near future.

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Correction: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models

Cited by 2 publications

References 1 publication

Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses

Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses

Gene therapy in inherited retinal degenerative diseases, a review

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