{"id":43588,"date":"2025-05-17T20:38:51","date_gmt":"2025-05-17T15:08:51","guid":{"rendered":"https:\/\/www.nextias.com\/ca\/?p=43588"},"modified":"2025-05-17T20:39:03","modified_gmt":"2025-05-17T15:09:03","slug":"gene-editing-therapy-used-to-cure-rare-disease","status":"publish","type":"post","link":"https:\/\/www.nextias.com\/ca\/current-affairs\/17-05-2025\/gene-editing-therapy-used-to-cure-rare-disease","title":{"rendered":"Gene Editing Therapy Used to Cure a Rare Disease"},"content":{"rendered":"\n<p><strong>Syllabus: GS3\/ Science &amp; Technology<\/strong><\/p>\n\n\n\n<p><strong>In News<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>In a historic medical breakthrough, scientists in the United States have, for the first time, successfully used a personalized<strong> CRISPR-based gene-editing therapy<\/strong> to treat a rare genetic disorder <strong>Carbamoyl Phosphate Synthetase 1 (CPS1) deficiency in an infant.<\/strong>\n<ul class=\"wp-block-list\">\n<li>CPS-1 Deficiency is a rare genetic metabolic disorder where the liver lacks an enzyme to convert toxic ammonia into urea, which is usually excreted through urine.<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>What is Gene Editing Therapy?<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Definition:\u00a0<\/strong>\n<ul class=\"wp-block-list\">\n<li>Gene editing therapy refers to the deliberate<strong> alteration of DNA sequences <\/strong>within the cells of an individual to treat or cure genetic diseases.\u00a0<\/li>\n\n\n\n<li>It involves modifying, deleting, or inserting specific genes at targeted locations in the genome to correct mutations or enhance cellular functions.<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li><strong>Types of Gene Editing Techniques:<\/strong>\n<ul class=\"wp-block-list\">\n<li><strong>CRISPR-Cas9:<\/strong> Most widely used tool; cuts DNA at specific sites using guide RNA and Cas9 enzyme<\/li>\n\n\n\n<li><strong>Zinc Finger Nucleases (ZFNs): <\/strong>Uses engineered proteins to bind and cut DNA<\/li>\n\n\n\n<li><strong>Base Editing: <\/strong>Alters a single nucleotide without breaking DNA strands<\/li>\n\n\n\n<li><strong>Prime Editing<\/strong>: Acts like a \u2018word processor\u2019 to insert, delete, or replace DNA sequences<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li><strong>Mechanism:\u00a0<\/strong><\/li>\n<\/ul>\n\n\n<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-large is-resized\"><img data-dominant-color=\"f0efed\" data-has-transparency=\"false\" loading=\"lazy\" decoding=\"async\" width=\"1024\" height=\"583\" src=\"https:\/\/wp-images.nextias.com\/cdn-cgi\/image\/format=auto\/ca\/uploads\/2025\/05\/What-is-Gene-Editing-Therapy-1024x583.png\" alt=\"What is Gene Editing Therapy\" class=\"not-transparent wp-image-43589\" style=\"--dominant-color: #f0efed; width:448px;height:auto\" srcset=\"https:\/\/wp-images.nextias.com\/cdn-cgi\/image\/format=auto\/ca\/uploads\/2025\/05\/What-is-Gene-Editing-Therapy-1024x583.png 1024w, https:\/\/wp-images.nextias.com\/cdn-cgi\/image\/format=auto\/ca\/uploads\/2025\/05\/What-is-Gene-Editing-Therapy-300x171.png 300w, https:\/\/wp-images.nextias.com\/cdn-cgi\/image\/format=auto\/ca\/uploads\/2025\/05\/What-is-Gene-Editing-Therapy-768x437.png 768w, https:\/\/wp-images.nextias.com\/cdn-cgi\/image\/format=auto\/ca\/uploads\/2025\/05\/What-is-Gene-Editing-Therapy-1536x874.png 1536w, https:\/\/wp-images.nextias.com\/cdn-cgi\/image\/format=auto\/ca\/uploads\/2025\/05\/What-is-Gene-Editing-Therapy.png 2048w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" \/><\/figure>\n<\/div>\n\n\n<h2 class=\"wp-block-heading\"><strong>Applications of Gene Editing Therapy<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Medicine:<\/strong> Treating genetic disorders like Sickle Cell Anaemia, Beta-Thalassemia, CPS-1 Deficiency.<\/li>\n\n\n\n<li>Engineering immune cells (CAR-T therapy) to target cancer<\/li>\n\n\n\n<li><strong>Agriculture:<\/strong> Developing disease-resistant, high-yield crops<\/li>\n\n\n\n<li><strong>Veterinary Science:<\/strong> Enhancing livestock traits<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>India\u2019s Progress in Gene Editing<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>CSIR-Institute of Genomics and Integrative Biology (IGIB) is developing indigenous <strong>CRISPR platform (IndiCRISPR).<\/strong><\/li>\n\n\n\n<li>The Department of Biotechnology (DBT) is funding genome-editing research under the National<strong> Biopharma Mission.<\/strong><\/li>\n\n\n\n<li><strong>Gene Therapy Guidelines (2020):<\/strong> Issued to streamline ethical clinical application.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Challenges\u00a0<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Safety issues:<\/strong> Off-target effects, immune reactions, unintended mutations.<\/li>\n\n\n\n<li><strong>Ethical concerns: <\/strong>\u00a0Germline editing (heritable changes), designer babies<\/li>\n\n\n\n<li><strong>Limited access:<\/strong> High cost, availability limited to developed nations<\/li>\n<\/ul>\n\n\n\n<p><strong>Source: IE<\/strong><\/p>\n","protected":false},"excerpt":{"rendered":"<h3><strong>In News<\/strong><\/h3>\n<li class=\"ms-5\">In a historic medical breakthrough, scientists in the United States have, for the first time, successfully used a personalized CRISPR-based gene-editing therapy to treat a rare genetic disorder Carbamoyl Phosphate Synthetase 1 (CPS1) deficiency in an infant.<\/li>\n<p><\/p>\n<h3><strong>Applications of Gene Editing Therapy<\/strong><\/h3>\n<li class=\"ms-5\">Medicine: Treating genetic disorders like Sickle Cell Anaemia, Beta-Thalassemia, CPS-1 Deficiency.<\/li>\n<li class=\"ms-5\">Engineering immune cells (CAR-T therapy) to target cancer<\/li>\n<li class=\"ms-5\">Agriculture: Developing disease-resistant, high-yield crops<\/li>\n<li class=\"ms-5\">Veterinary Science: Enhancing livestock traits<\/li>\n<p><a href=\"https:\/\/www.nextias.com\/ca\/current-affairs\/17-05-2025\/gene-editing-therapy-used-to-cure-rare-disease\" class=\"btn btn-primary btn-sm float-end\">Read\u00a0More<\/a><\/p>\n","protected":false},"author":15,"featured_media":0,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[21],"tags":[],"class_list":["post-43588","post","type-post","status-publish","format-standard","hentry","category-current-affairs"],"acf":[],"jetpack_featured_media_url":"","_links":{"self":[{"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts\/43588","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/users\/15"}],"replies":[{"embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/comments?post=43588"}],"version-history":[{"count":3,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts\/43588\/revisions"}],"predecessor-version":[{"id":43604,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/posts\/43588\/revisions\/43604"}],"wp:attachment":[{"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/media?parent=43588"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/categories?post=43588"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.nextias.com\/ca\/wp-json\/wp\/v2\/tags?post=43588"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}