{"id":271111,"date":"2023-11-25T20:12:00","date_gmt":"2023-11-25T14:42:00","guid":{"rendered":"https:\/\/forumias.com\/blog\/?p=271111"},"modified":"2023-11-25T20:12:00","modified_gmt":"2023-11-25T14:42:00","slug":"casgevy-how-gene-therapy-will-revolutionize-treatment-of-sickle-cell-disease","status":"publish","type":"post","link":"https:\/\/forumias.com\/blog\/casgevy-how-gene-therapy-will-revolutionize-treatment-of-sickle-cell-disease\/","title":{"rendered":"Casgevy: How gene therapy will revolutionize treatment of sickle cell disease"},"content":{"rendered":"

Source: <\/b>The post is based on the article <\/span>\u201c<\/b>Casgevy: <\/b>How gene therapy will revolutionize treatment of sickle cell disease\u201d <\/b>published in<\/span> \u201cIndian Express\u201d on 25th November 2023<\/b><\/p>\n

Why in the News?<\/h2>\n

The UK drug regulator has approved a gene therapy called Casgevy<\/strong> for the cure of sickle cell<\/strong><\/span> disease <\/strong>and thalassaemia<\/strong>.<\/span><\/p>\n

What is Casgevy?<\/h2>\n\n\n\n\n\n\n\n\n
Specifications<\/b><\/td>\nDetails<\/b><\/td>\n<\/tr>\n
What is it?<\/b><\/td>\nIt is a<\/span> CRISPR Cas9 based gene-edited therapy developed for the treatment of S<\/span>ickle cell<\/span> disease<\/span> and Thalassaemia.<\/span><\/td>\n<\/tr>\n
Working<\/b><\/td>\n1) Casgevy is a one-time treatment.
\n<\/span>2) The treatment involves removing stem cells out of bone marrow from a patient\u2019s blood using a process called apheresis that filters out the blood for different components.
\n<\/span>3) CRISPR gene editing technology is then used in a laboratory to edit the faulty gene.
\n<\/span>4) The edited cells are then infused back into the patient, allowing the body to produce functioning haemoglobin.<\/span><\/td>\n<\/tr>\n
Target Gene<\/b><\/td>\n– A gene called <\/span>BCL11A <\/b>which is crucial for switching from foetal to adult haemoglobin is targeted by the therapy.
\n<\/span>– Note:<\/b> Foetal haemoglobin which is naturally present in everyone at birth, does not carry the same abnormalities as adult haemoglobin.
\n<\/span>– The therapy uses the body\u2019s own mechanisms to start producing more of this foetal haemoglobin alleviating the symptoms of the two conditions.<\/span><\/td>\n<\/tr>\n
Challenges<\/b><\/td>\n1) <\/span>High Cost <\/b>is one of the biggest limitations of gene therapies.
\n<\/span>2) <\/span>Absence of local manufacturing facilitie<\/b>s<\/strong> which means that the harvested blood stem cells have to be sent across countries.<\/span><\/td>\n<\/tr>\n
Significance<\/b><\/td>\nThis is the first licensed therapy in the world based on the gene editing technology Crispr-Cas9<\/strong> that earned its innovators a Nobel Prize in 2020.<\/span><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n

What is CRISPR Cas9 Technology?<\/h2>\n

Click Here to read<\/b><\/a><\/p>\n

What is Sickle Cell Disease?<\/h2>\n
\"Sickle
Source: Indian Express<\/figcaption><\/figure>\n\n\n\n\n\n\n\n
Specifications<\/b><\/td>\nDetails<\/b><\/td>\n<\/tr>\n
What is it<\/b><\/td>\nIt is an inherited red blood cell disorder that affect hemoglobin<\/strong>, a protein that carries oxygen through the body.\u00a0<\/span><\/td>\n<\/tr>\n
Characteristics of the disease<\/b><\/td>\n1) Normally, red blood cells are disc-shaped and flexible enough to move easily through the blood vessels.
\n<\/span>2) In sickle cell disease, red blood cells become crescent- or \u201csickle\u201d-shaped due to a genetic mutation<\/strong>.
\n<\/span>3) Such a shape makes the flow of hemoglobin through the blood vessels difficult and can block blood flow<\/strong> to the rest of the body.
\n<\/span>4) The blocked blood flow through the body can lead to serious problems, including stroke, eye problems, infections, and episodes of pain called pain crises.<\/span><\/td>\n<\/tr>\n
Treatment<\/b><\/td>\nBone Marrow Transplant<\/span><\/td>\n<\/tr>\n
Initiatives<\/b><\/td>\nNational Sickle Cell Anaemia Elimination Mission (NSCEM<\/span><\/a>)<\/span><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n

What is T<\/b>halassaemia?<\/b><\/h2>\n

1) T<\/span>halassaemia <\/span>is a chronic and genetic blood disorder<\/strong> due to which a patient\u2019s body cannot make enough hemoglobin leading to anemia<\/strong>.<\/span><\/p>\n

2) This disease is passed from parents to children through genes and people suffering from it require blood transfusions to survive.<\/span><\/p>\n

UPSC Syllabus: Science and Technology<\/strong><\/p>\n","protected":false},"excerpt":{"rendered":"

Source: The post is based on the article \u201cCasgevy: How gene therapy will revolutionize treatment of sickle cell disease\u201d published in \u201cIndian Express\u201d on 25th November 2023 Why in the News? The UK drug regulator has approved a gene therapy called Casgevy for the cure of sickle cell disease and thalassaemia. What is Casgevy? Specifications… Continue reading Casgevy: How gene therapy will revolutionize treatment of sickle cell disease<\/span><\/a><\/p>\n","protected":false},"author":10317,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"jetpack_post_was_ever_published":false,"footnotes":""},"categories":[1566,1738],"tags":[11872],"class_list":["post-271111","post","type-post","status-publish","format-standard","hentry","category-daily-factly-articles","category-science-and-technology-daily-factly-articles","tag-9pm-daily-factly","entry"],"jetpack_featured_media_url":"","views":{"total":6,"cached_at":"","cached_date":1700951661},"jetpack_sharing_enabled":true,"_links":{"self":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts\/271111","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/users\/10317"}],"replies":[{"embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/comments?post=271111"}],"version-history":[{"count":0,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/posts\/271111\/revisions"}],"wp:attachment":[{"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/media?parent=271111"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/categories?post=271111"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/forumias.com\/blog\/wp-json\/wp\/v2\/tags?post=271111"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}