Viral-Vector-Delivered Anti-Angiogenic Therapies to the Eye

Koponen, Sanna; Kokki, Emmi; Kinnunen, Kati; Ylä‐Herttuala, Seppo

doi:10.3390/pharmaceutics13020219

Cited by 12 publications

(14 citation statements)

References 121 publications

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“…The practice of transferring genetic material to remove, replace, repair, or introduce a gene to treat disorders is known as gene therapy [75]. Viral vectors, naked DNA, and nonviral vectors such as nanoparticles, microinjection, electroporation, sonoporation, and iontophoresis might all be used to deliver genes.…”

Section: Gene-based Ocular Delivery Systemsmentioning

confidence: 99%

Recent Strategies for Ocular Drug Delivery: Promises and Challenges

El-Kamel¹,

Ashour²

2023

Advanced Drug Delivery Systems

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Ocular diseases include various anterior and posterior segment diseases. Due to the unique anatomy and physiology of the eye, efficient ocular drug delivery is a great challenge to researchers. The emerging nanoscience is playing an important role in the development of novel strategies for ocular disease management. Various active molecules have been designed to associate with nanocarriers to overcome ocular barriers and interact with certain ocular tissues. In this chapter, highlights will be made on barrier to intraocular delivery, general pathways for ocular absorption, and factors affecting intraocular bioavailability. The recent attempts of nanotechnology for treating anterior and posterior ocular diseases will be explored. This will include nanomicelles, nanoparticles, nanosuspensions, vesicular systems, in situ gel, dendrimers, contact lenses, implants, microneedles, and cell-based delivery systems. In addition, gene-based ocular delivery systems will be discussed. In this chapter, we will also provide a comprehensive overview of drug-device combinations used for ocular diseases such as glaucoma, dry eye disease, infections, and inflammations. Furthermore, drug delivery devices for ocular surgeries are discussed. Finally, challenges and future prospective of ocular delivery systems will be explored.

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Section: Gene-based Ocular Delivery Systemsmentioning

confidence: 99%

Recent Strategies for Ocular Drug Delivery: Promises and Challenges

El-Kamel¹,

Ashour²

2023

Advanced Drug Delivery Systems

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“…Moreover, delivering DNA to the retina using AAV has been useful in clinical trials since 2008 [ 15 ]. For example, the FDA approved the first gene therapy treatment for ocular disease in 2017 for the treatment of inherited biallelic RPE65 mutation-associated retinal dystrophy by delivering AAV2-mediated VN subretinally [ 42 ]. This therapy allows hope for the recessive forms of the disorder.…”

Section: Indications For Subretinal Injectionsmentioning

confidence: 99%

“…In clinical trials, the treatment has been considered safe, lacking severe adverse effects. Nonetheless, larger clinical trials are needed [ 42 ].…”

Section: Indications For Subretinal Injectionsmentioning

confidence: 99%

Subretinal Injection Techniques for Retinal Disease: A Review

Irigoyen

Alonso

Sanchez-Molina

et al. 2022

JCM

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Inherited retinal dystrophies (IRDs) affect an estimated 1 in every 2000 people, this corresponding to nearly 2 million cases worldwide. Currently, 270 genes have been associated with IRDs, most of them altering the function of photoreceptors and retinal pigment epithelium. Gene therapy has been proposed as a potential tool for improving visual function in these patients. Clinical trials in animal models and humans have been successful in various types of IRDs. Recently, voretigene neparvovec (Luxturna®) has been approved by the US Food and Drug Administration for the treatment of biallelic mutations in the RPE65 gene. The current state of the art in gene therapy involves the delivery of various types of viral vectors into the subretinal space to effectively transduce diseased photoreceptors and retinal pigment epithelium. For this, subretinal injection is becoming increasingly popular among researchers and clinicians. To date, several approaches for subretinal injection have been described in the scientific literature, all of them effective in accessing the subretinal space. The growth and development of gene therapy give rise to the need for a standardized procedure for subretinal injection that ensures the efficacy and safety of this new approach to drug delivery. The goal of this review is to offer an insight into the current subretinal injection techniques and understand the key factors in the success of this procedure.

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“…Gene augmentation strategy provides a long-term persistent expression of secreted therapeutic proteins to deal with non-genetic retinal disease, such as AMD. The interim results of Regenxbio from phase I, open-label dose increasing trial assessing the efficacy and safety of the subretinal injection of a novel AAV8 vector (RGX-314) encoding a soluble anti-VEGF monoclonal antibody fragment (NCT03066258) were encouraging (92). The expression levels of the protein were observable at 1 month in a dose-dependent manner, with sustained expression observed at 6 months in patients treated at a dose of 6e10 vector genome (vg) per eye.…”

Section: Gene Augmentationmentioning

confidence: 99%

Advancing precision medicines for ocular disorders: Diagnostic genomics to tailored therapies

et al. 2022

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Successful sequencing of the human genome and evolving functional knowledge of gene products has taken genomic medicine to the forefront, soon combining broadly with traditional diagnostics, therapeutics, and prognostics in patients. Recent years have witnessed an extraordinary leap in our understanding of ocular diseases and their respective genetic underpinnings. As we are entering the age of genomic medicine, rapid advances in genome sequencing, gene delivery, genome surgery, and computational genomics enable an ever-increasing capacity to provide a precise and robust diagnosis of diseases and the development of targeted treatment strategies. Inherited retinal diseases are a major source of blindness around the world where a large number of causative genes have been identified, paving the way for personalized diagnostics in the clinic. Developments in functional genetics and gene transfer techniques has also led to the first FDA approval of gene therapy for LCA, a childhood blindness. Many such retinal diseases are the focus of various clinical trials, making clinical diagnoses of retinal diseases, their underlying genetics and the studies of natural history important. Here, we review methodologies for identifying new genes and variants associated with various ocular disorders and the complexities associated with them. Thereafter we discuss briefly, various retinal diseases and the application of genomic technologies in their diagnosis. We also discuss the strategies, challenges, and potential of gene therapy for the treatment of inherited and acquired retinal diseases. Additionally, we discuss the translational aspects of gene therapy, the important vector types and considerations for human trials that may help advance personalized therapeutics in ophthalmology. Retinal disease research has led the application of precision diagnostics and precision therapies; therefore, this review provides a general understanding of the current status of precision medicine in ophthalmology.

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Viral-Vector-Delivered Anti-Angiogenic Therapies to the Eye

Cited by 12 publications

References 121 publications

Recent Strategies for Ocular Drug Delivery: Promises and Challenges

Recent Strategies for Ocular Drug Delivery: Promises and Challenges

Subretinal Injection Techniques for Retinal Disease: A Review

Advancing precision medicines for ocular disorders: Diagnostic genomics to tailored therapies

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