{"id":85635,"date":"2018-12-09T11:45:19","date_gmt":"2018-12-09T19:45:19","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2018\/12\/scientists-may-have-halted-blindness-caused-by-this-rare-genetic-disease"},"modified":"2018-12-09T11:45:19","modified_gmt":"2018-12-09T19:45:19","slug":"scientists-may-have-halted-blindness-caused-by-this-rare-genetic-disease","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2018\/12\/scientists-may-have-halted-blindness-caused-by-this-rare-genetic-disease","title":{"rendered":"Scientists May Have Halted Blindness Caused by This Rare Genetic Disease"},"content":{"rendered":"

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D esigner therapies are treatments tailored to a specific disease, and nowhere is the need greater for new therapies than in a group of nervous system disorders, known as \u201cneurodegenerative diseases.\u201d<\/p>\n

Many of these diseases are common and well-known, such as Alzheimer\u2019s<\/a> or Parkinson\u2019s<\/a> disease. However, some are very rare, genetic disorders that are the consequence of a defective gene. In all these diseases, a mutant protein that misfolds causes the degeneration and death of neurons. One effective therapeutic strategy is to prevent the rogue protein from ever being made.<\/p>\n

Spinocerebellar ataxia type 7 (SCA7) is one such disease in which nerves in different parts of the brain, including the eye, degenerate, which leads to blindness and difficulty walking, speaking, and balancing. SCA7 is dominantly inherited \u2014 which means that you just need one bad copy of the mutation to cause disease. The disease occurs when a short section of DNA that encodes ataxin-7 gene is erroneously repeated<\/a> \u2014 like a word in a book printed two or three times. In this case, three chemical units of the DNA sequence \u2014 C-A-G \u2014 are repeated over and over.<\/p>\n

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D esigner therapies are treatments tailored to a specific disease, and nowhere is the need greater for new therapies than in a group of nervous system disorders, known as \u201cneurodegenerative diseases.\u201d Many of these diseases are common and well-known, such as Alzheimer\u2019s or Parkinson\u2019s disease. However, some are very rare, genetic disorders that are the [\u2026]<\/p>\n","protected":false},"author":396,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,412,47],"tags":[],"class_list":["post-85635","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-genetics","category-neuroscience"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/85635","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/396"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=85635"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/85635\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=85635"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=85635"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=85635"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}