Ocular gene therapy: introduction to the special issue

Ali, Robin R.

doi:10.1038/gt.2011.189

Cited by 10 publications

(3 citation statements)

References 9 publications

(6 reference statements)

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“…Gene therapy has emerged as a transformative strategy in the field of ocular therapeutics, offering unprecedented opportunities to address genetic mutations and disorders and potentially superior effects compared to conventional treatments. [ 140 ] Nucleic acids (e.g., siRNA, mRNA, etc.) are negatively charged and hydrophilic, preventing their penetration through the cellular membrane.…”

Section: Modifying Carrier Physicochemical Properties For Enhanced Oc...mentioning

confidence: 99%

Long‐Acting Ocular Injectables: Are We Looking In The Right Direction?

Dang,

Shoichet

2023

Advanced Science

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The complex anatomy and physiological barriers of the eye make delivering ocular therapeutics challenging. Generally, effective drug delivery to the eye is hindered by rapid clearance and limited drug bioavailability. Biomaterial‐based approaches have emerged to enhance drug delivery to ocular tissues and overcome existing limitations. In this review, some of the most promising long‐acting injectables (LAIs) in ocular drug delivery are explored, focusing on novel design strategies to improve therapeutic outcomes. LAIs are designed to enable sustained therapeutic effects, thereby extending local drug residence time and facilitating controlled and targeted drug delivery. Moreover, LAIs can be engineered to enhance drug targeting and penetration across ocular physiological barriers.

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Section: Modifying Carrier Physicochemical Properties For Enhanced Oc...mentioning

confidence: 99%

Long‐Acting Ocular Injectables: Are We Looking In The Right Direction?

Dang,

Shoichet

2023

Advanced Science

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“…The lack of cell division in the retina also allows for the use of non-integrating vector systems, thereby reducing the risk of insertional mutagenesis and oncogenesis. 58 …”

Section: Gene Therapymentioning

confidence: 99%

Neovascular Age-Related Macular Degeneration (nAMD): A Review of Emerging Treatment Options

Tan¹,

Ngo²,

Chay³

et al. 2022

OPTH

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Neovascular age-related macular degeneration (nAMD) is a common world-wide cause of visual loss. Intravitreal anti-vascular endothelial growth factor (anti-VEGF) agents are an effective means to treat nAMD and reduce its impact on vision compared to either sham treatment or photodynamic therapy. Currently, the approved anti-VEGF drugs include ranibizumab, aflibercept and brolucizumab. In addition, bevacizumab, used as an off-label drug, and has been shown to be effective in treating nAMD. While anti-VEGF agents are effective, its limitations include the requirement for frequent, often monthly injections, and the need for long-term treatment of nAMD. These present significant burdens on the healthcare system and on the patients. In addition, reviews of patients with nAMD treated with anti-VEGF have reported deterioration of vision over time with progression of geographic atrophy. These limitations are partly addressed by exploring different treatment regimens that reduce the frequency of treatments. Newer anti-VEGF drugs have been shown in Phase III clinical trials to have injection intervals as long as 12 or even 16 weeks for a proportion of patients. There is research on newer drugs that affect other pathways, such as the angiopoietin pathway, which may impact nAMD by extending the treatment interval and reducing the burden of treatment. Other measures include the use of sustained-release implants that release the drug regularly over a period of time, and can be refilled periodically, as well as hydrogel platforms that serve to release the drug. The use of biosimilars will also serve to reduce the cost of treatment for nAMD. A new frontier of gene therapy, primarily targeting genes involved in the transduction of retinal cells to produce anti-VEGF proteins intraocularly, also opens a new avenue of therapeutic approaches that can be used for treatment. This review paper will discuss both current treatment options and the newer treatments under development.

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“…AAV vectors have recently attracted much attention as gene therapy vehicles in clinical trials for the treatment of inherited retinal diseases, e.g. Leber's congenital amaurosis [23]; however, its value as an efficient vector for corneal gene therapy is controversially discussed in the literature. While it has been described that AAV shows no or little efficiency in transducing corneal cells [24], other groups report successful AAV gene therapy using novel pseudotyping strategies [25].…”

Section: Genetic Modification Of Corneal Allografts Prior To Transplamentioning

confidence: 99%

Gene Therapy Approaches to Prevent Corneal Graft Rejection: Where Do We Stand?

2013

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Cornea transplantation (penetrating keratoplasty) is the most frequently performed transplant procedure in humans. Despite advances in microsurgery and immunosuppressive treatment protocols, a significant number of corneal grafts still undergo immune-mediated allograft rejection. Topical treatment with corticosteroids is currently the gold standard and while this treatment is effective in many corneal transplant patients, it is much less effective in ‘high-risk' patients with previous episodes of neovascularisation or graft rejection. Therefore, alternative approaches such as genetic modification of donor corneas are needed to prevent corneal transplant rejection. Cornea transplantation holds the unique advantage in that gene therapy can be used to modify allografts ex vivo prior to transplantation. Many preclinical studies using local (and systemic) gene transfer have been performed to date and many different gene transfer vehicles (gene therapy vectors) and therapeutic strategies (immunomodulatory or graft-protective) have been investigated to prevent corneal allograft rejection. The most recent gene therapy applications to prevent corneal allograft rejection will be reviewed in this article. Moreover, it will be discussed why the development of clinical trials for the genetic modification of corneal grafts prior to transplantation is lagging behind of those for the treatment of inherited retinal diseases.

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Ocular gene therapy: introduction to the special issue

Cited by 10 publications

References 9 publications

Long‐Acting Ocular Injectables: Are We Looking In The Right Direction?

Long‐Acting Ocular Injectables: Are We Looking In The Right Direction?

Neovascular Age-Related Macular Degeneration (nAMD): A Review of Emerging Treatment Options

Gene Therapy Approaches to Prevent Corneal Graft Rejection: Where Do We Stand?

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