Gene Therapy to Prevent Blindness luke Caligan #2, Quinlyn Phan #20

How does Gene Therapy work? Gene Therapy is when a missing cells are replaced by normal cells to correct genetic disorders. Our main point in Gene Therapy is to Prevent blindness. This blindness is caused by many gene disorders and electronic radiation. The “machine” to cure blindness was only developed last year.

Some people doing Gene Therapy

These gene disorders include Type One/Two Diabetes, Retinal Detachment, Cataracts, and Tay-Sach Disease. Type One/Two Diabetes you go high on sugar which causes blurriness too your eye. Retinal Detachment is when one specific part of your eye is unattached. Cataracts blocks your eye with built up bacteria/germs. Tay-Sach Disease is when the embryo in the mother's womb has a nerve which damages the eye of the baby.

The therapy should work like this: Gene therapy Electrocardiograms (Pods that are taped on someone’s head that can record their brain wave activities and sight levels) are used to the patient’s forehead and pores. Then, treatments in the form of a liquid substance is used. The rear part of the eye is where conditions such as preoperative diabetic retinopathy and age-related macular degeneration develop, and they involve a substance called vascular endothelial growth factor, which stimulates the growth of blood vessels.

Most researchers use gene therapy to prevent blindness, as said. Gene therapy methods to prevent blindness is still being developed and being tested on animals. Since it is still currently developing, to get gene therapy to prevent Leber's Congenital Amaurosis, then you would have to get eye injections (Needle shots, which can be quite painful). These methods are quite fresh, because they were invented about last year. If you want gene testing right now, most likely chances you can get gene therapy, but it could cost millions of dollars.

Scientists have been trying to inhibit the growth factor using gene therapy, but delivering drugs to the back of the eye currently requires an injection (Which can STING). Researchers developed a gene delivery system with a peptide called penetratin. Which has shown good permeability in the eye, and a synthetic polymer called poly-amidoamine that has previously been used in drug delivery. Before testing it on regular humans, they started to use rats as test subjects. The results showed that the complex, when applied as an eye drop, rapidly moved from the eye’s surface to its rear inner lining. The findings demonstrate the method’s potential for delivering gene therapy to treat several eye diseases, which one day, we hope it will be used on human beings!


Created with images by Giuliamar - "eye eyelashes face" • UC Davis College of Engineering - "SU13_KentLeach_MT_Edit (28 of 65)" • Dizzy-one - "Evening blur"

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