Swiss researchers have developed a gene-editing technique to potentially cure Stargardt disease, an inherited eye disease causing progressive vision loss in children and young adults. The technique, which uses an adenine base editor, has shown promising results in mice and nonhuman primates, but further human trials are needed.
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A research team from the Institute of Molecular and Clinical Ophthalmology, Switzerland, has developed a technique to edit genes, to demonstrate how it could be used to perhaps cure Stargardt disease.
Stargardt disease, also known as Stargardt macular degeneration, is an inherited eye disease that leads to progressive vision loss, commonly in children and young adults.
It is caused by mutations in the ABCA4 gene, which affects how the body utilises Vitamin A. This results in the buildup of a yellowish-brown pigment called lipofuscin in the macula, which is the central part of the retina responsible for sharp, central vision.
The disease leads to the slow loss of central vision in both eyes, which can worsen over time. People with Stargardt disease may experience gray, black, or hazy spots in the center of their vision, light sensitivity, and color blindness.
The buildup of lipofuscin in the retina disrupts normal vision, leading to legal blindness in many cases.
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At Present, there is no cure for Stargardt disease. |
Swiss researchers have developed a gene-editing technique for treating Stargardt disease, by using an adenine base editor, which can make precise DNA changes, has shown promising results in mice and nonhuman primates.
Gene editing holds great potential for treating hereditary eye diseases. Researchers believe it could provide a cure for diseases like Stargardt, which were previously untreatable. While this gene therapy is still in early stages, the success in animal models offers hope that similar results could be achieved in humans. However, human trials are essential to determine its long-term effectiveness.
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PRACTICE QUESTION Q.Consider the following statements about Stargardt disease: Statement 1: Stargardt disease results from mutations in the ABCA4 gene. Statement 2: It is characterized by a buildup of lipofuscin in the retina. Which of the following is correct? A) Both Statement 1 and Statement 2 are correct. B) Statement 1 is correct but Statement 2 is incorrect. C) Statement 1 is incorrect but Statement 2 is correct. D) Both Statement 1 and Statement 2 are incorrect. Answer: A Explanation: Stargardt disease is caused by mutations in the ABCA4 gene, which leads to a buildup of lipofuscin in retinal cells, particularly in the macula, leading to vision impairment. |
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