Advancement of Gene Therapy Treatment for Ophthalmic Diseases

Advancement of Gene Therapy Treatment for Ophthalmic Diseases
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It is supposed that 80% of the perceived information by the brain originates comes from the visual system, and any abnormality in the visual system may significantly lower life quality. There are many causes of visual dysfunction; the most important of them are glaucoma, cataracts, and age-related macular degeneration (AMD), causing 69% of global blindness.Though there is a high incidence of glaucoma and AMD but little information about it due to the unknown underlying pathologies, as experiment-based studies of glaucoma and AMD are limited in humans, animal models play an important role in investigating the underlying molecular mechanisms and new therapeutic research. Appropriateanimal models such as rats, mice, monkeys, and zebrafish, enable the identification of new genes involved in animal and human pathology and explain the genetic relationships between different genes, such as causative and modifier genes. The availability of animal models has established valuable and effective ocular level microenvironments that mimic human cells and help better understand disease patterns. For example, different dry eye animal models such as evaporation, lacrimal secretion insufficiency, neuronal dysfunction, and environmental stress are linked with different etiologic factors. [caption align="aligncenter" width="512"] Animal Models for Treating Ophthalmic Diseases[/caption] Other models of animals can be categorized as having multiple disease-causing factors. For example, several models of glaucoma, such as animal models, cell cultures, and post-mortem eyes, have been studied with many advantages and disadvantages. Through the animal model, the interaction between visual and animal organisms can be well understood. Hence, compared to the cell cultures experiment, the animal models present more similar physiological responses in glaucoma patients.The recent predicaments and clinical trials in treating ophthalmic diseasesAnimal models typify the disease with high precision in glaucoma research. Due to pre-clinical experiments, animal models replicate some lesions such as damage in retinal ganglion cell (RGC) axons, some changes in the anterior segment, death of RGC, alterations due to ocular hypertension (OH), and some alterations in the head of the optic nerve (ON) and the lamina cribrosa (LC) and being cost-effective during practical. An immense number of pre-clinical studies have been conducted on rodents in the field of glaucoma. Different animal models, such as monkeys, rats, mice, dogs, and pigsare in use with their limitations and advantages.Innovation of gene therapy treatment for treating ophthalmic diseases
  1. TurboKnockout technology
Turboknockout, which expedites your ophthalmic research, is a cutting-edge technology that is more advanced than ES cell-mediated gene targeting. It shortened the time duration of CRISPR-Cas9 from 1 year to 6 months. Compared to CRISPR-Cas9, it offers more accurate gene targeting with a large size target region up to 300kb. The turbo knockout gene targeting services can be used in complex gene modeling
  1. CRISPR technology
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. CRISPR sequences were first discovered in the Escherichia coli genome, and it was found that they function as part of an RNA-based adaptive immune system that targets and destroys genetic parasites.An endonuclease known as CRISPR-associated protein (Cas) cuts foreign DNA, allowing it to be incorporated into the host genome. Invasion DNA can only be cleaved when there is a protospacer adjacent motif (PAM) surrounding the targeted sequence. Researchers studying CRISPR have adapted it for use as a tool for genetic modification of the target host genome. Due to its simplicity and versatility, CRISPR/Cas9 has recently become a popular genome-editing tool. [caption align="aligncenter" width="512"] CRISPR technology[/caption]
  1. Mouse models that mimic ophthalmic diseases
Many diseases should be investigated through animal models, such as chronic retinal degenerative disease; retinitis pigmentosa has been continually lacking effective treatment. But its animal model, including the ribozyme technique, is still under investigation. Yet a large leap forward step has been taken in animal models in treating a specific type of retinal degeneration, Leber's congenital amaurosis.The evasion of the subconjunctival-episcleral clearance mechanism might be the main reason of the eye, in animal research, drug delivery through the microneedle hybrid technique has resulted in 80-fold greater intraocular bioavailability of the drug with a 3-fold increased duration of action. Therefore, in animal models, pre-experimen is critical, the structural characteristics of drugs or prodrugs that help in ocular penetration, retina, and choroid may be enhanced accordingly. How do these technologies speed up the search for ways to cure ophthalmic diseases? Genetically engineered viruses are injected into the space between the white of the eye and the vascular layer using a small needle. Researchers used CRISPR gene editing to partially restore vision in legally blind patients: Shots - Health News Doctors used a gene-editing tool directly in patients' eyes for the first time. The experiment restored color and shape perception to these patients with impaired vision. Cyagen advanced gene therapy solution for effectively treating ophthalmic diseasesCyagen is providing gene therapy solutions of advanced level for treating patients through genetic engineering. Choose the best one, and choose Cyagen for accurate results and within the minimum time limitFor more information, don't hesitate to get in touch with Cyagen at: