{"id":94820,"date":"2023-04-14T14:27:00","date_gmt":"2023-04-14T18:27:00","guid":{"rendered":"https:\/\/www.shortform.com\/blog\/?p=94820"},"modified":"2023-04-18T13:27:45","modified_gmt":"2023-04-18T17:27:45","slug":"treatment-of-genetic-disorders","status":"publish","type":"post","link":"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/","title":{"rendered":"Treatment of Genetic Disorders: The Challenges &#038; Advances"},"content":{"rendered":"\n<p>What is gene therapy? Are genetic disorders curable? How far are we from a future where gene therapy is mainstream? <\/p>\n\n\n\n<p>Gene therapy\u2014using <a href=\"https:\/\/www.shortform.com\/blog\/are-we-still-evolving\/\">genetic engineering<\/a> to fix damaged or disease-causing genes\u2014offers promising treatments for many genetic disorders. However, we are still far from making gene therapy mainstream because of the high costs of research and development. <\/p>\n\n\n\n<p>Here&#8217;s a look at the challenges and advances in the treatment of genetic disorders. <\/p>\n\n\n\n<!--more-->\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-gene-therapy-to-cure-genetic-diseases\"><strong>Gene Therapy to Cure Genetic Diseases<\/strong><\/h2>\n\n\n\n<p>Gene therapy is a process that uses viruses to replace mutated, illness-causing DNA in the body\u2019s cells with healthy DNA. The injected virus containing the healthy DNA attaches itself to the patient\u2019s cells and causes them to start replicating that healthy DNA, eliminating the mutation that was causing the disease.&nbsp;<\/p>\n\n\n\n<p>Another type of treatment using genes is gene editing, which allows scientists to edit DNA within cells. An editing protein called CRISPR-Cas9 locates the DNA that needs to be edited and snips out the portion of it that\u2019s causing problems. Its ability to locate the affected DNA also makes it an excellent diagnostic tool for rapidly detecting diseases, including cancer and viral infections such as COVID-19. Gene editing is a newer technology than gene therapy, and research is underway to discover more editing proteins to expand our options in what diseases we treat and how.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\"><strong>Challenges and Advances in Gene Therapy<\/strong><\/h3>\n\n\n\n<p>Breakthroughs continue to be made in the fields of gene therapy and gene editing, but the <a href=\"https:\/\/www.rarediseaseadvisor.com\/conferences\/wodc-2022\/cell-gene-therapy-breakthroughs-outpace-healthcare-system-experts-say\/\" target=\"_blank\" rel=\"noreferrer noopener\">high costs of research are placing a bottleneck on development<\/a>. While it already costs, on average, over $2.5 billion to bring a new drug to market, the unique nature of gene therapy treatments <a href=\"https:\/\/www.evernorth.com\/articles\/challenges-in-bringing-gene-therapies-to-market\" target=\"_blank\" rel=\"noreferrer noopener\">raises the average cost to $5 billion<\/a>.\u00a0<\/p>\n\n\n\n<p>These high development costs translate into extremely high costs for the treatment of genetic disorders as well. In nations with nationalized healthcare, <a href=\"https:\/\/www.nytimes.com\/2023\/01\/24\/health\/gene-therapies-cost-zolgensma.html\" target=\"_blank\" rel=\"noreferrer noopener\">governments are struggling to foot the bills<\/a> of these therapies that can cost millions of dollars per treatment. In America, <a href=\"https:\/\/www.webmd.com\/a-to-z-guides\/news\/20230215\/life-saving-gene-therapies-cost-millions\" target=\"_blank\" rel=\"noreferrer noopener\">gene therapies are often covered by insurance<\/a> but the free market nature of the healthcare industry leaves insurers unable to negotiate for lower prices.\u00a0<\/p>\n\n\n\n<p>Despite the costs, gene therapy continues to advance, with <a href=\"https:\/\/themedicinemaker.com\/business-regulation\/the-biggest-cgt-breakthroughs-through-the-eyes-of-our-2022-power-list\" target=\"_blank\" rel=\"noreferrer noopener\">recent developments including<\/a> cures for spinal muscular atrophy, inherited retinal disease, and large B-cell lymphoma. <a href=\"https:\/\/www.nature.com\/articles\/d41586-022-04210-5\" target=\"_blank\" rel=\"noreferrer noopener\">Further research may yield cures<\/a> for Duchenne muscular dystrophy and hemophilia. Gene editing recently saw <a href=\"https:\/\/www.usnews.com\/news\/health-news\/articles\/2023-01-26\/new-breakthrough-could-speed-gene-therapy-research\" target=\"_blank\" rel=\"noreferrer noopener\">a breakthrough that could rapidly accelerate further research<\/a>: Human-derived proteins called zinc fingers may provide a safer alternative to <a href=\"https:\/\/www.shortform.com\/blog\/crispr-gene-editing-technology\/\">CRISPR<\/a> treatment, which uses bacteria-derived proteins that run the risk of triggering an immune response.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>What is gene therapy? Are genetic disorders curable? How far are we from a future where gene therapy is mainstream? Gene therapy\u2014using genetic engineering to fix damaged or disease-causing genes\u2014offers promising treatments for many genetic disorders. However, we are still far from making gene therapy mainstream because of the high costs of research and development. Here&#8217;s a look at the challenges and advances in the treatment of genetic disorders.<\/p>\n","protected":false},"author":7,"featured_media":23931,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_jetpack_memberships_contains_paid_content":false,"footnotes":""},"categories":[16,160],"tags":[929],"class_list":["post-94820","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-health","category-science","tag-life-force","","tg-column-two"],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v24.3 (Yoast SEO v24.3) - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Treatment of Genetic Disorders: The Challenges &amp; Advances - Shortform Books<\/title>\n<meta name=\"description\" content=\"Gene therapy offers promising treatments for many genetic disorders. However, we are far from a future where gene therapy is mainstream.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Treatment of Genetic Disorders: The Challenges &amp; Advances\" \/>\n<meta property=\"og:description\" content=\"Gene therapy offers promising treatments for many genetic disorders. However, we are far from a future where gene therapy is mainstream.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/\" \/>\n<meta property=\"og:site_name\" content=\"Shortform Books\" \/>\n<meta property=\"article:published_time\" content=\"2023-04-14T18:27:00+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2023-04-18T17:27:45+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/s3.amazonaws.com\/wordpress.shortform.com\/blog\/wp-content\/uploads\/2021\/01\/cell-dna-science-lab.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"845\" \/>\n\t<meta property=\"og:image:height\" content=\"531\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"Darya Sinusoid\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"Darya Sinusoid\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/\"},\"author\":{\"name\":\"Darya Sinusoid\",\"@id\":\"https:\/\/www.shortform.com\/blog\/#\/schema\/person\/0421cce75bc249b11e2517b3a91f9c46\"},\"headline\":\"Treatment of Genetic Disorders: The Challenges &#038; Advances\",\"datePublished\":\"2023-04-14T18:27:00+00:00\",\"dateModified\":\"2023-04-18T17:27:45+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/\"},\"wordCount\":433,\"commentCount\":0,\"publisher\":{\"@id\":\"https:\/\/www.shortform.com\/blog\/#organization\"},\"image\":{\"@id\":\"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/www.shortform.com\/blog\/wp-content\/uploads\/2021\/01\/cell-dna-science-lab.jpg\",\"keywords\":[\"Life Force\"],\"articleSection\":[\"Health\",\"Science\"],\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"CommentAction\",\"name\":\"Comment\",\"target\":[\"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/#respond\"]}]},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/\",\"url\":\"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/\",\"name\":\"Treatment of Genetic Disorders: The Challenges & Advances - Shortform Books\",\"isPartOf\":{\"@id\":\"https:\/\/www.shortform.com\/blog\/#website\"},\"primaryImageOfPage\":{\"@id\":\"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/#primaryimage\"},\"image\":{\"@id\":\"https:\/\/www.shortform.com\/blog\/treatment-of-genetic-disorders\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/www.shortform.com\/blog\/wp-content\/uploads\/2021\/01\/cell-dna-science-lab.jpg\",\"datePublished\":\"2023-04-14T18:27:00+00:00\",\"dateModified\":\"2023-04-18T17:27:45+00:00\",\"description\":\"Gene therapy offers promising treatments for many genetic disorders. 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