{"id":38273,"date":"2023-11-22T10:45:35","date_gmt":"2023-11-22T05:15:35","guid":{"rendered":"https:\/\/vajiramandravi.com\/current-affairs\/?p=38273"},"modified":"2025-04-23T09:28:29","modified_gmt":"2025-04-23T03:58:29","slug":"gene-therapy","status":"publish","type":"post","link":"https:\/\/vajiramandravi.com\/current-affairs\/gene-therapy\/","title":{"rendered":"Gene Therapy for Sickle Cell Anaemia: Significance for Indian Patients"},"content":{"rendered":"<h2>Why in News?<\/h2>\n<ul>\n<li>USA&#8217;s Victoria Gray is the world\u2019s first sickle cell anaemia (SCA) patient to recover with the revolutionary gene-editing therapy.<\/li>\n<li>Gray underwent a clinical trial in 2017 for the drug Casgevy, which uses the innovative gene-editing tool CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats and associated protein 9).<\/li>\n<\/ul>\n<h2>What\u2019s in Today\u2019s Article?<\/h2>\n<ul>\n<li>Why in News?<\/li>\n<li>What is Sickle Cell Anaemia (SCA)?<\/li>\n<li>How the Pathbreaking Gene Therapy for SCA Works?<\/li>\n<li>Significance of this Pathbreaking Gene Therapy for SCA for India<\/li>\n<li>Concerns Regarding the Gene Therapy for SCA and Way Ahead<\/li>\n<\/ul>\n<h2>What is Sickle Cell Anaemia (SCA)?<\/h2>\n<p><img decoding=\"async\" src=\"https:\/\/vajiramandravi.s3.us-east-1.amazonaws.com\/media\/editor_images\/2023\/11\/22\/13\/37\/24\/655db6bcae9d5a059930abe2_OI.PNG\" alt=\"\" \/><\/p>\n<ul>\n<li>A genetic disease, SCA arises from mutations in haemoglobin-carrying genes, causing red blood cells to assume a crescent shape.<\/li>\n<li>This potentially obstructs blood flow and leads to severe pain, organ damage, strokes, and other complications.<\/li>\n<li>Globally, the current remedy for the disease is limited to bone marrow transplants (which must come from a closely matched donor and carries a risk of rejection).<\/li>\n<\/ul>\n<h2>How the Pathbreaking Gene Therapy for SCA Works?<\/h2>\n<p><img decoding=\"async\" src=\"https:\/\/vajiramandravi.s3.us-east-1.amazonaws.com\/media\/editor_images\/2023\/11\/22\/13\/37\/26\/655db6beae9d5a059930abe3_OI.PNG\" alt=\"\" \/><\/p>\n<ul>\n<li>The CRISPR-Cas9 technique basically involves modifying the patient\u2019s DNA, specifically targeting and replacing the faulty haemoglobin gene with a healthy one.<\/li>\n<li>To do this, stem cells are taken out of the bone marrow, edited in a laboratory and then infused back into the patient.<\/li>\n<li>This restores normal haemoglobin function, offering a potential cure for a lifetime.<\/li>\n<li>About 29 patients were administered the treatment (along with Gray), of whom 28 have no pain and will be followed up for lasting cure.\n<ul>\n<li>The therapy also works for patients suffering from transfusion-dependent \u00df-thalassemia.<\/li>\n<\/ul>\n<\/li>\n<li>Recently, the UK approved (became first country to do so)the therapy &#8211; called Casgevy, as it has been the only permanent, innovative and first-of-its-kind gene-editing treatment option.<\/li>\n<\/ul>\n<h2>Significance of this Pathbreaking Gene Therapy for SCA for India<\/h2>\n<p><img decoding=\"async\" src=\"https:\/\/vajiramandravi.s3.us-east-1.amazonaws.com\/media\/editor_images\/2023\/11\/22\/13\/37\/28\/655db6c0ae9d5a059930abe4_OI.PNG\" alt=\"\" \/><\/p>\n<ul>\n<li>The breakthrough is of particular significance to India, which has the second-highest disease burden of SCA globally after African countries.<\/li>\n<li>An estimated 30,000-40,000 children in India are born with the disorder every year.<\/li>\n<li>In 2019, a nationwide screening by the Ministry of Tribal Affairs and the Indian Council of Medical Research (ICMR) found the disease to be prevalent in 8.75% of those screened.<\/li>\n<li>Latest data shows that one in 86 births among the Scheduled Tribe (ST) population is affected by SCA, with higher rates in central, western and southern India.<\/li>\n<li>In her 2023-24 Budget presentation, the Finance Minister of India had said that the government aimed to eliminate the disease by 2047.<\/li>\n<\/ul>\n<figure class=\"media\">\n<div data-oembed-url=\"https:\/\/www.youtube.com\/watch?v=BOjceonSiTY\">\n<div><iframe src=\"https:\/\/www.youtube.com\/embed\/BOjceonSiTY\" frameborder=\"0\" allowfullscreen=\"allowfullscreen\"><\/iframe><\/div>\n<\/div>\n<\/figure>\n<h2>Concerns Regarding the Gene Therapy for SCA and Way Ahead<\/h2>\n<ul>\n<li>While Vertex and CRISPR Therapeutics (the manufacturers) have yet to set a price for the gene-editing therapy in the UK, at the moment it is expected to exceed Rs 1 crore in India, which could potentially limit access.<\/li>\n<li>However, government intervention and subsidies, along with crowdfunding and philanthropy, can enable access to cure.<\/li>\n<li>The UK\u2019s approval would inspire Indian researchers to develop their own innovative therapies of CRISPR like they did with CAR-T for blood cancer, ultimately making the treatment more affordable.<\/li>\n<\/ul>\n<hr \/>\n<h3>Q1) What are genetic disorders?<\/h3>\n<p>Genetic disorders are a category of diseases that includes certain types of birth defects, chronic diseases, developmental problems, and sensory deficits that are inherited from one or both parents.<\/p>\n<h3>Q2) Why is sickle cell anaemia common in tribes of India?<\/h3>\n<p>The tribal areas in India were endemic to malaria for many years leading to an evolutionary trait &#8211; their red blood cells were becoming sickle-shaped. This led to their high susceptibility to sickle cell disease, including alpha-thalassemia.<\/p>\n<hr \/>\n<p><strong>Source:<\/strong> <a href=\"https:\/\/indianexpress.com\/article\/health-wellness\/uk-gene-therapy-sickle-cell-anaemia-indian-patients-9036706\/\" target=\"_blank\" rel=\"nofollow noopener\"><u>UK approves gene therapy for sickle cell anaemia. Why it offers hope for Indian patients<\/u><\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>The drug Casgevy uses the innovative gene-editing tool CRISPR-Cas9, which targets and replaces the faulty haemoglobin gene with a healthy one to treat Sickle Cell disease<\/p>\n","protected":false},"author":5,"featured_media":38274,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[18],"tags":[],"class_list":{"0":"post-38273","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-upsc-mains-current-affairs","8":"no-featured-image-padding"},"acf":[],"_links":{"self":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/posts\/38273","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/users\/5"}],"replies":[{"embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/comments?post=38273"}],"version-history":[{"count":0,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/posts\/38273\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/media\/38274"}],"wp:attachment":[{"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/media?parent=38273"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/categories?post=38273"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/vajiramandravi.com\/current-affairs\/wp-json\/wp\/v2\/tags?post=38273"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}