The Evolution of Retinal Gene Therapy: From Clinical Trials to Clinical Practice

Pennesi, Mark E.; Schlecther, Catherine L.

doi:10.1016/j.ophtha.2019.12.003

Cited by 10 publications

(5 citation statements)

References 4 publications

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“…Subretinal injection is being more commonly used in the field of ophthalmology. The first subretinally injected drug was Voretigen Neparvovec (Luxturna), which was approved by American Food and Drug Administration in the 2017 [ 16 ]. This medicament was used as therapy for RPE65-mediated retinal dystrophy – being untreated, it would progress into the completed blindness.…”

Section: Discussionmentioning

confidence: 99%

“…This medicament was used as therapy for RPE65-mediated retinal dystrophy – being untreated, it would progress into the completed blindness. Initial trials showed promise into improvement of functional vision, which was before untreatable [ 16 ]. Compared to intravitreal or suprachoroidal injections, SI allows for drug delivery directly into retina cells, where intravitreal injections rely on diffusion from intravitreal cavity, resulting in lower absorption due to the presence of impermeable internal limiting membrane [ 17 – 20 ]…”

Section: Discussionmentioning

confidence: 99%

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Sharper vision, steady hands: can robots improve subretinal drug delivery? Systematic review

Łajczak,

Nawrat

2024

J Robotic Surg

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Subretinal injection (SI) is a novel drug delivery method, directly to retina for treatment of various eye disease. However, manual injection requires surgical experience and precision due to physiological factors. Robots offer solution to this issue, by reducing hand tremor and increased accuracy. This systematic review analyzes current status on robot-assisted SI compared to conventional techniques. Systematic search across 5 databases was conducted to identify studies comparing manual and robot-assisted SI procedures. Extracted data included robotic systems, complications, and success rates. Four studies, including one human trial, two ex vivo porcine eye studies, and one artificial eye model study were included in the synthesis. The findings show early advantages of robot-assisted SI. Compared to traditional interventions, robot procedures result in reduced tremor, what potentially lowers the risk of complications, including retinal tears and reflux. The first in-human randomized trial showed encouraging results, with no significant differences in surgical times or complications between robot-assisted and manual SI. However, major limitation of robot-assisted procedures is longer procedure time. Robot-assisted SI holds promise by offering increased precision and stability, reducing human error and potentially improving clinical outcomes. Challenges include cost, availability, and learning curve. Overall, early stage of robot-assisted SI suggests advantages in precision, complication reduction, and potentially improved drug delivery. Further research in human randomized trials is needed to fully assess its full-scale clinical application.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Sharper vision, steady hands: can robots improve subretinal drug delivery? Systematic review

Łajczak,

Nawrat

2024

J Robotic Surg

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“…In recent years, gene therapy has been the focus for treating IRDs [ 27 ]. Commonly, the two routes of delivery are by subretinal or intravitreal injection, which maximizes transduction to the target cells in the eye ( Figure 3 ).…”

Section: Gene Therapy For Irdsmentioning

confidence: 99%

Base and Prime Editing in the Retina—From Preclinical Research toward Human Clinical Trials

Yee

Wert

2022

IJMS

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Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of diseases that are one of the leading causes of vision loss in young and aged individuals. IRDs are mainly caused by a loss of the post-mitotic photoreceptor neurons of the retina, or by the degeneration of the retinal pigment epithelium. Unfortunately, once these cells are damaged, it is irreversible and leads to permanent vision impairment. Thought to be previously incurable, gene therapy has been rapidly evolving to be a potential treatment to prevent further degeneration of the retina and preserve visual function. The development of clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9) base and prime editors have increased the capabilities of the genome editing toolbox in recent years. Both base and prime editors evade the creation of double-stranded breaks in deoxyribonucleic acid (DNA) and the requirement of donor template of DNA for repair, which make them advantageous methods in developing clinical therapies. In addition, establishing a permanent edit within the genome could be better suited for patients with progressive degeneration. In this review, we will summarize published uses of successful base and prime editing in treating IRDs.

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“…Such gene therapy approach is expensive regarding the very few number of RP patients affected by one specific gene (e.g. 425 000 $ per eye for LUXTURNA TM (Pennesi and Schlecther 2020)). Of note, gene mutations causing RP are not known in 30% of nonsyndromic and 50% of autosomal-dominant RP patients (Hartong et al 2006;Verbakel et al 2018).…”

Section: Current Strategies Under Developmentmentioning

confidence: 99%

Photoreceptor Cell Replacement Using Pluripotent Stem Cells: Current Knowledge and Remaining Questions

Monville

Goureau

M’Barek

2022

Cold Spring Harb Perspect Med

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Retinal degeneration is an increasing global burden without cure for the majority of patients. Once retinal cells have degenerated, vision is permanently lost. Different strategies have been developed in the recent years to prevent retinal degeneration or to restore sight (e.g., gene therapy, cell therapy and electronic implants). Herein, we present current treatment strategies with a focus on cell therapy for photoreceptor replacement using human pluripotent stem cells. We will describe the state of the art and discuss obstacles and limitations observed in preclinical animal models as well as future directions to improve graft integration and functionality.

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The Evolution of Retinal Gene Therapy: From Clinical Trials to Clinical Practice

Cited by 10 publications

References 4 publications

Sharper vision, steady hands: can robots improve subretinal drug delivery? Systematic review

Sharper vision, steady hands: can robots improve subretinal drug delivery? Systematic review

Base and Prime Editing in the Retina—From Preclinical Research toward Human Clinical Trials

Photoreceptor Cell Replacement Using Pluripotent Stem Cells: Current Knowledge and Remaining Questions

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