BLOG: New blog focuses on tenets, strategies in ophthalmic gene-based treatments
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In recent decades, ophthalmology has been a leader in exploring cutting-edge technologies for combating blinding diseases, and innovative gene-based strategies have shown promising results.
This blog hopes to discuss the latest news in this field but also topics pertinent to the ophthalmic community, including the surgical methods that allow treatment delivery and the issues surrounding them.
From the time that the human DNA sequence was deciphered, genetic discovery has identified more than 270 genes known to cause rare forms of hereditary eye disease, according to RetNet, and a battery of predisposing genetic markers for more common diseases, such as age-related macular degeneration and glaucoma, has also been established. This genetic information has given physicians and scientists the tools to explore gene-based therapeutic strategies for ocular diseases that still represent a significant unmet medical need.
Strategies in genetic medicine stem from the basic theory that replacing, altering or adding specific genetic sequences can reverse, prevent or delay the natural history of the target disease and therefore can have a significant impact on the patient’s quality of life.
This blog looks to inform the reader on the basic tenets of genomic medicine, the strategies that currently exist in gene-based therapeutics, the surgical modalities needed to deliver them, possible surgical complications and the diseases that new treatments hope to combat. Additionally, other topics will be presented, such as the need for appropriate endpoints in clinical development, news on clinical trial safety and the role of the vitreoretinal surgeon in genomic medicine. We will also feature guest bloggers from the vitreoretinal field, the point of view of patient advocates and exciting new developments from retinal research scientists. Today, gene-based medicine is no longer an aspiration or a fantasy but a reality. Gaining insight on these innovative genomic medicines will help physicians better inform their patients on treatment options or the clinical trials they could participate in.
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