Recent advances in the management and understanding of macular degeneration

Bahadorani, Sepehr; Singer, Michael

doi:10.12688/f1000research.10998.1

Cited by 18 publications

(7 citation statements)

References 32 publications

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“…Gyroscope's Therapeutics' FOCUS trial utilizing GT-005 is a gene therapy designed to target patients with dry AMD. Dry AMD accounts for 85-90% of all AMD cases [39]. Unlike nAMD, there are currently no effective therapies available for dry AMD.…”

Section: Gt-005mentioning

confidence: 99%

Review of gene therapies for age-related macular degeneration

Khanani

Thomas

Aziz

et al. 2022

Eye

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Gene therapies aim to deliver a therapeutic payload to specified tissues with underlying protein deficiency. Since the 1990s, gene therapies have been explored as potential treatments for chronic conditions requiring lifetime care and medical management. Ocular gene therapies target a range of ocular disorders, but retinal diseases are of particular importance due to the prevalence of retinal disease and the current treatment burden of such diseases on affected patients, as well as the challenge of properly delivering these therapies to the target tissue. The purpose of this review is to provide an update on the most current data available for five different retinal gene therapies currently undergoing clinical trials for use against age-related macular degeneration (AMD) and the development of novel delivery routes for the administration of such therapies. Research has been performed and compiled from PubMed and the select authors of this manuscript on the treatment and effectiveness of five current retinal gene therapies: Luxturna, ADVM-022, RGX-314, GT-005, and HMR59. We present the available data of current clinical trials for the treatment of neovascular and dry age-related macular degeneration with different AAV-based gene therapies. We also present current research on the progress of developing novel routes of administration for ocular gene therapies. Retinal gene therapies offer the potential for life-changing treatment for chronic conditions like age-related macular degeneration with a single administration. In doing so, gene therapies change the landscape of treatment options for these chronic conditions for both patient and provider.

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Section: Gt-005mentioning

confidence: 99%

Review of gene therapies for age-related macular degeneration

Khanani

Thomas

Aziz

et al. 2022

Eye

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“…[73] The dry form is the most common, affecting 85-90 percent of the population. [74] Drusen, tiny yellow deposits found between the RPE and the Bruch's membrane on an ocular coherence tomography (OCT) scan indicate dry AMD. These deposits are detected beneath the retina and can range in size.…”

Section: • Age-related Macular Degeneration (Amd)mentioning

confidence: 99%

Theranostic Applications of Nanomaterials for Ophthalmic Applications

Kajaria¹,

Yashwant²,

Kevin³

2021

International Journal of Scientific Advances

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The development of theranostic applications of nanomaterials has presented researchers with a new form of diagnosing and treating diseases, such as cancer, Diabetic Retinopathy, and Age-Related Macular Degeneration. Theranostic have allowed for a bridge between conventional medicine and personalized medicine with the use of nanomaterials. The applications of nanomaterials within medicine (nanomedicine) have changed the diagnosis, monitoring, control, treatment, and prevention of diseases. The effects of the biological interaction of nanomaterials within the human body must be examined further to understand the efficacy, toxicity, and biodistribution. The use of theranostic application of nanomaterials has gained attention in the field of Ophthalmology. The use of theranostic nanomaterials to reduce current treatments, complications, diagnosis, reduce the effects and treat a visually threatening disease is appealing. The benefit of treating ocular diseases with theranostic will allow for better visibility and targeted treatment.

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“…Age-related Macular Degeneration (AMD) is categorised as the primary cause of blindness affecting people above the age of 55 years and it is estimated to infringe 196 million people worldwide by the year 2020 [9]. AMD can be labelled as wet AMD (neovascular choroidal) accounting for 10-15% of the cases and dry AMD (geographic atrophy) computing for 80-85% of the total cases [3,10]. RPE cells are situated below the photoreceptors which perform essential functions as mentioned earlier; these cells are majorly affected by AMD, making it a complex degenerative disease caused as a result of a low-grade inflammation in the retina leading to deterioration of RPE and Bruch's membrane.…”

Section: Age-related Macular Degenerationmentioning

confidence: 99%

“…Although the success rate is low as the RPE cells are previously damaged and surgery might lead to the further removal of healthy cells [12]. Stem cell therapy is a potential treatment for AMD as it can persuade improvement of visual perception [10].…”

Section: Age-related Macular Degenerationmentioning

confidence: 99%

Ocular Stem Cells to Treat Retinal and Corneal Disorders

Chatterji¹,

Mehvash²,

Roma³

2018

TOSCJ

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Background:According to WHO, 285 million people are visually impaired out of which, 39 million are classified as blind and the remaining 246 million people have low vision which comprises of moderate vision impairment and severe vision impairment. Therapies to treat major disorders leading to visual impairment like Age-related Macular Degeneration (AMD), Stargardt’s Disease (STGD), Retinitis Pigmentosa (RP) and corneal scarring are required.In the last decade, many advances have been made to treat these disorders using stem cell therapy. For corneal damage by accidental burns, scarring or limbal stem cell deficiencies which can lead to partial or total blindness, are treated with a risky intervention like keratoplasty. To overcome issues like graft rejection caused by keratoplasty as well as have a better outcome, limbal stem cell therapy has been introduced. Similarly, Retinal Pigment Epithelium (RPE) is a supporting tissue essential in nutrient transport, production of growth factors, phagocytosis of the photoreceptors and retinol cycling.Discussion and Conlusion:Degeneration of this monolayer causes many diseases that have no prevailing treatment; however, research is being carried out to replace this simple epithelial monolayer primarily with an autologous source of cells and currently using stem cells. This review discusses the advances made in the field of ocular stem cell therapy with regards to development, cultivation and novel methods used to deliver these cells to replace the corneal and retinal epithelium as a new standard for treatment.

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Recent advances in the management and understanding of macular degeneration

Cited by 18 publications

References 32 publications

Review of gene therapies for age-related macular degeneration

Review of gene therapies for age-related macular degeneration

Theranostic Applications of Nanomaterials for Ophthalmic Applications

Ocular Stem Cells to Treat Retinal and Corneal Disorders

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