{"id":823,"date":"2024-08-10T17:23:53","date_gmt":"2024-08-10T17:23:53","guid":{"rendered":"https:\/\/clickmen.us\/news\/?p=823"},"modified":"2024-08-10T17:23:53","modified_gmt":"2024-08-10T17:23:53","slug":"first-gene-editing-therapy-could-cure-disorder","status":"publish","type":"post","link":"https:\/\/clickmen.us\/news\/first-gene-editing-therapy-could-cure-disorder\/","title":{"rendered":"First gene-editing therapy could cure disorder"},"content":{"rendered":"

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\"KirthanaKirthana Balachandran<\/span><\/div>\n<\/div>\n

Kirthana Balachandran was diagnosed when she was three months old and is now studying to be a doctor<\/figcaption><\/p>\n<\/figure>\n
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The first therapy that uses gene-editing is to be offered on the NHS in a \u201crevolutionary breakthrough\u201d for patients.<\/p>\n

It will be used as a potential cure for the blood disorder beta thalassaemia.<\/p>\n

Stem cells which make blood will be extracted, reprogrammed to correct the condition and returned to the patient’s body. <\/p>\n

It could spare them needing a blood transfusion, every three to five weeks, for life.<\/p>\n<\/div>\n

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People with beta thalassaemia struggle to produce enough haemoglobin, which is the protein in red blood cells that carries oxygen around the body.<\/p>\n

It is a genetic disease that is passed down through families and caused by defects in the body’s instructions for manufacturing haemoglobin. <\/p>\n

It can leave people severely tired, weak, and short of breath and also cuts life expectancy.<\/p>\n

Kirthana Balachandran, 21, was diagnosed when she was only three months old and gets muscle pain and back pain and can have palpitations when walking uphill.<\/p>\n

\u201cThe idea of depending on transfusions for quite literally the rest of my life is daunting, I constantly worry about the future,\u201d she says.<\/p>\n

The idea of gene-editing is you only need to do it once.<\/p>\n<\/div>\n

How the gene-editing works<\/h2>\n<\/p>\n
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It relies on a tool called Crispr, which won the Nobel Prize for Chemistry in 2020. It is essentially a satnav connected to a pair of scissors \u2013 one part targets the right section of DNA and the other performs the edit.<\/p>\n

You might think this technology is being used to repair the genetic defect, but it is actually cuter than that. <\/p>\n

Instead it relies on the fact your body makes different types of haemoglobin before and after birth.<\/p>\n

When we are still in the womb, our bodies use “fetal haemoglobin” to pull oxygen out of our mother’s bloodstream.<\/p>\n

Once we are born, a genetic switch is flipped and we start making “adult haemoglobin”. <\/p>\n

Crucially, it is only the adult form of haemoglobin that is affected by beta thalassaemia.<\/p>\n

So the therapy disables the switch – named BCL11A \u2013 so the adult body starts making fetal haemoglobin once again. <\/p>\n

In order to do this, the stem cells that manufacture red blood cells in our spongy bone marrow are harvested.<\/p>\n

These cells are sent to the lab where the genetic switch is targeted.<\/p>\n

The next stage is unpleasant. It takes a course of chemotherapy to kill off the old stem cells that were producing broken haemoglobin, before the new ones can be put in.<\/p>\n<\/div>\n

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Abdul-Qadeer Akhtar, 28 and from Hemel Hempstead, took part in clinical trials in 2020.<\/p>\n

He said the treatment was \u201cchallenging\u201d and \u201ctough\u201d but since having it, he has been \u201chealthier and more active\u201d.<\/p>\n

\u201c[I] have even taken up boxing, I can travel more freely now, which is fantastic, I\u2019m eager to embrace life to the fullest.\u201d<\/p>\n

Data shows that out of 52 patients given the treatment, 49 did not need another blood transfusion for at least a year of monitoring. The approach is so new that the long-term effects are still unknown. <\/p>\n

Kirthana says: \u201cWith gene therapy, I would potentially have a cure, and not have to have my three-weekly transfusions.<\/p>\n

\u201cIt would be a life-changing treatment.\u201d<\/p>\n<\/div>\n

A \u00a31.6m price tag?<\/h2>\n<\/p>\n
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The National Institute of Health and Care Excellence medicines watchdog has approved the therapy after assessing the costs and benefits of what it calls a \u201cpotential cure\u201d.<\/p>\n

A deal has been struck which means NHS England is paying less than the official price of \u00a31.6m per patient.<\/p>\n

It is estimated that 460 people over the age of 12 would be eligible if they wanted the therapy, and it will be offered at seven specialist centres \u201cwithin weeks\u201d.<\/p>\n

Amanda Pritchard, the NHS chief executive, said: \u201cThis is a historic moment for people living with beta thalassaemia with a potential cure for those facing this debilitating disorder now available on the NHS.\u201d<\/p>\n

Beta thalassemia mainly affects people of Mediterranean, south Asian, south-east Asian and Middle Eastern backgrounds.<\/p>\n

Previously the only alternative to blood transfusions was a stem cell transplant, but this was rare because it required a very close tissue match from a donor. <\/p>\n

Casgevy, developed by the company Vertex, is the first approved therapy to use Crispr-technology.<\/p>\n

Romaine Maharaj, executive director of the UK Thalassaemia Society, said \u201cwe stand on the brink of a revolutionary breakthrough\u201d and \u201cit is a beacon of hope\u201d.<\/p>\n

Negotiations are still under way to see if the same therapy can be used on the NHS for another genetic disease affecting haemoglobin – sickle cell anaemia. <\/p>\n<\/div>\n<\/div>\n

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[ad_1] Kirthana Balachandran Kirthana Balachandran was diagnosed when she was three months old and is now studying to be a doctor The first therapy that uses gene-editing is to be offered on the NHS in a \u201crevolutionary breakthrough\u201d for patients. It will be used as a potential cure for the blood disorder beta thalassaemia. Stem […] More<\/a><\/p>\n","protected":false},"author":1,"featured_media":824,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[135],"tags":[],"adace-sponsor":[],"class_list":{"0":"post-823","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-health"},"_links":{"self":[{"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/posts\/823","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/comments?post=823"}],"version-history":[{"count":1,"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/posts\/823\/revisions"}],"predecessor-version":[{"id":837,"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/posts\/823\/revisions\/837"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/media\/824"}],"wp:attachment":[{"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/media?parent=823"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/categories?post=823"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/tags?post=823"},{"taxonomy":"adace-sponsor","embeddable":true,"href":"https:\/\/clickmen.us\/news\/wp-json\/wp\/v2\/adace-sponsor?post=823"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}