{"id":677697,"date":"2025-06-28T13:15:22","date_gmt":"2025-06-28T10:15:22","guid":{"rendered":"https:\/\/buradabiliyorum.com\/en\/gene-therapy-reduces-stroke-risk-factors-in-sickle-cell-disease-patients\/"},"modified":"2025-06-28T13:15:22","modified_gmt":"2025-06-28T10:15:22","slug":"gene-therapy-reduces-stroke-risk-factors-in-sickle-cell-disease-patients","status":"publish","type":"post","link":"https:\/\/buradabiliyorum.com\/en\/gene-therapy-reduces-stroke-risk-factors-in-sickle-cell-disease-patients\/","title":{"rendered":"Gene therapy reduces stroke risk factors in sickle cell disease patients"},"content":{"rendered":"<div id=\"ez-toc-container\" class=\"ez-toc-v2_0_85 counter-hierarchy ez-toc-counter ez-toc-custom ez-toc-container-direction\">\n<p class=\"ez-toc-title\" style=\"cursor:inherit\">Table of Contents<\/p>\n<label for=\"ez-toc-cssicon-toggle-item-6a3dca511ddf2\" class=\"ez-toc-cssicon-toggle-label\"><span class=\"\"><span class=\"eztoc-hide\" style=\"display:none;\">Toggle<\/span><span class=\"ez-toc-icon-toggle-span\"><svg style=\"fill: #dd3333;color:#dd3333\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" class=\"list-377408\" width=\"20px\" height=\"20px\" viewBox=\"0 0 24 24\" fill=\"none\"><path d=\"M6 6H4v2h2V6zm14 0H8v2h12V6zM4 11h2v2H4v-2zm16 0H8v2h12v-2zM4 16h2v2H4v-2zm16 0H8v2h12v-2z\" fill=\"currentColor\"><\/path><\/svg><svg style=\"fill: #dd3333;color:#dd3333\" class=\"arrow-unsorted-368013\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"10px\" height=\"10px\" viewBox=\"0 0 24 24\" version=\"1.2\" baseProfile=\"tiny\"><path d=\"M18.2 9.3l-6.2-6.3-6.2 6.3c-.2.2-.3.4-.3.7s.1.5.3.7c.2.2.4.3.7.3h11c.3 0 .5-.1.7-.3.2-.2.3-.5.3-.7s-.1-.5-.3-.7zM5.8 14.7l6.2 6.3 6.2-6.3c.2-.2.3-.5.3-.7s-.1-.5-.3-.7c-.2-.2-.4-.3-.7-.3h-11c-.3 0-.5.1-.7.3-.2.2-.3.5-.3.7s.1.5.3.7z\"\/><\/svg><\/span><\/span><\/label><input type=\"checkbox\"  id=\"ez-toc-cssicon-toggle-item-6a3dca511ddf2\" checked aria-label=\"Toggle\" \/><nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/buradabiliyorum.com\/en\/gene-therapy-reduces-stroke-risk-factors-in-sickle-cell-disease-patients\/#Improving_blood_flow_in_the_brain_with_gene_therapy\" >Improving blood flow in the brain with gene therapy<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/buradabiliyorum.com\/en\/gene-therapy-reduces-stroke-risk-factors-in-sickle-cell-disease-patients\/#Gene_therapy_is_comparable_or_better_than_other_treatments_when_it_comes_to_brain_blood_flow\" >Gene therapy is comparable or better than other treatments when it comes to brain blood flow<\/a><\/li><\/ul><\/nav><\/div>\n<div>\n<div class=\"article-gallery lightGallery\">\n<div data-thumb=\"https:\/\/scx1.b-cdn.net\/csz\/news\/tmb\/2025\/gene-therapy-improves.jpg\" data-src=\"https:\/\/scx2.b-cdn.net\/gfx\/news\/hires\/2025\/gene-therapy-improves.jpg\" data-sub-html=\"Corresponding author Akshay Sharma, MBBS, MSc, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy talks to St. Jude patient enrolled on the reported clinical trial, Corey Luellen. Credit: St. Jude Children's Research Hospital\">\n<figure class=\"article-img\">\n            <img loading=\"lazy\" decoding=\"async\" src=\"https:\/\/scx1.b-cdn.net\/csz\/news\/800a\/2025\/gene-therapy-improves.jpg\" alt=\"Gene therapy improves blood flow in the brain in patients with sickle cell disease\" title=\"Corresponding author Akshay Sharma, MBBS, MSc, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy talks to St. Jude patient enrolled on the reported clinical trial, Corey Luellen. Credit: St. Jude Children's Research Hospital\" width=\"800\" height=\"530\"\/><figcaption class=\"text-darken text-low-up text-truncate-js text-truncate mt-3\">\n                Corresponding author Akshay Sharma, MBBS, MSc, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy talks to St. Jude patient enrolled on the reported clinical trial, Corey Luellen. Credit: St. Jude Children&#8217;s Research Hospital<br \/>\n            <\/figcaption><\/figure>\n<\/p><\/div>\n<\/div>\n<p>Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children&#8217;s Research Hospital. Many people with sickle cell disease experience increased brain ischemia, where oxygen is not delivered properly to brain tissues, potentially leading to strokes. A part of the risk for these events comes from increased blood flow speed in the brain. Findings from three patients in a gene therapy clinical trial showed that gene therapy treatment significantly improved blood flow in the brain. These results demonstrate that people with these risk factors may benefit from gene therapy and should be considered for future clinical trials of gene therapy. The findings were <a rel=\"nofollow\" target=\"_blank\" href=\"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ajh.27757\" target=\"_blank\">published<\/a> in the <i>American Journal of Hematology<\/i>.<\/p>\n<p>&#8220;We saw that after gene therapy, elevated blood flow speed in the brain came down to normal levels,&#8221; said corresponding author Akshay Sharma, MBBS, MSc, St. Jude Department of Bone Marrow Transplantation &amp; Cellular Therapy. &#8220;This is the closest physiological evidence we have that gene therapy could be effective for patients with neurovascular disease who are at risk of or have had a stroke.&#8221;<\/p>\n<h2><span class=\"ez-toc-section\" id=\"Improving_blood_flow_in_the_brain_with_gene_therapy\"><\/span>Improving blood flow in the brain with gene therapy<span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p>A small but significant percentage of patients with sickle cell disease are at an increased risk of stroke due to the condition&#8217;s impact on blood flow in the brain. The crescent &#8220;sickle&#8221; shape of red blood cells characteristic of sickle cell disease cannot move through small blood vessels, including those in the brain. When those vessels become clogged, that region in the brain does not receive enough oxygen. To compensate for the lack of oxygen delivery, the body amps up the speed of blood flow, which increases the number of red blood cells moving through the brain and the total amount of available oxygen. This results in a decrease in the time oxygen molecules have to leave the red blood cells and enter the brain tissue, which can ultimately lead to brain ischemia. Brain ischemia is when a region of the brain is greatly deprived of oxygen, creating a significant risk factor for stroke, which can result in long-term damage.<\/p>\n<p>&#8220;You can think of red blood cells filled with oxygen like a bus filled with people,&#8221; Sharma said. &#8220;If the bus is going too fast, passengers can&#8217;t get off the bus, and oxygen is not delivered. However, if the bus slows down so passengers can safely hop off, as h<a href=\"https:\/\/buradabiliyorum.com\/en\/category\/download-scripts-themes-apps\/\" data-internallinksmanager029f6b8e52c=\"9\" title=\"Download Scripts &amp; Themes &amp; Apps\" target=\"_blank\" rel=\"noopener\">app<\/a>ens when hemoglobin levels rise, then oxygen gets properly delivered to the brain tissues.&#8221;<\/p>\n<p>The researchers measured gene therapy&#8217;s effect on the flow of blood in the brain using Magnetic Resonance Imaging (MRI). The study imaged the brains of three patients with sickle cell disease, before gene therapy and at one and two years after the treatment. Each patient&#8217;s brain blood flow improved significantly, decreasing anywhere from 22% to 43%, reaching mostly normal levels, which appeared stable over time.<\/p>\n<h2><span class=\"ez-toc-section\" id=\"Gene_therapy_is_comparable_or_better_than_other_treatments_when_it_comes_to_brain_blood_flow\"><\/span>Gene therapy is comparable or better than other treatments when it comes to brain blood flow<span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p>Results from the study compare well to previous studies measuring the impact of other treatments for sickle cell disease, including the drug hydroxyurea or blood transfusions. Hydroxyurea, the most common treatment for sickle cell disease, has only a small effect on brain blood flow, and while blood transfusions have a stronger positive impact on brain blood flow, the effect is transient because the patient must continually receive new transfusions for it to last. The researchers found gene therapy has a more substantial and long-lasting protective effect on the brain than either of these treatments.<\/p>\n<p>Bone marrow transplants also normalize brain blood flow over the long term. While gene therapy and bone marrow transplantation were not compared directly in the study, the results suggest both treatments produce a similar return to normal blood flow in the brain that is durable over time.<\/p>\n<p>This study of three patients provides preliminary evidence for gene therapy&#8217;s effect on stroke risk and requires follow-up studies to confirm the result. However, it adds to a growing body of evidence that gene therapy should be considered as a treatment option to protect brain health in patients with sickle cell disease.<\/p>\n<p>The study provides support for new clinical trials for sickle cell disease gene therapy to include patients at risk for stroke. Historically, given the high-risk nature of their disease, these individuals have been excluded from gene therapy trials.<\/p>\n<p>&#8220;We now have emerging data to at least evaluate the efficacy of gene therapy in patients with a risk of or history of stroke,&#8221; Sharma said. &#8220;Until now, we only had one option that had a long-term impact on blood flow in the brain: bone marrow transplantation. But now we may also have gene therapy as another viable method to protect against neurovascular disease in people with sickle cell disease.&#8221;<\/p>\n<div class=\"article-main__more p-4\">\n<p><strong>More information:<\/strong><br \/>\n                                                    Akshay Sharma et al, Normalization of Cerebral Hemodynamics After Gene Therapy in Adults With Sickle Cell Disease, <i>American Journal of Hematology<\/i> (2025). <a rel=\"nofollow\" target=\"_blank\" data-doi=\"1\" href=\"https:\/\/dx.doi.org\/10.1002\/ajh.27757\" target=\"_blank\">DOI: 10.1002\/ajh.27757<\/a><\/p>\n<\/p><\/div>\n<div class=\"d-inline-block text-medium my-4\">\n\t\t\t\t\t\t\t\t\t\t\t\tProvided by<br \/>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\tSt. Jude Children&#8217;s Research Hospital<br \/>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a rel=\"nofollow\" target=\"_blank\" class=\"icon_open\" href=\"http:\/\/www.stjude.org\" target=\"_blank\" rel=\"nofollow\"><br \/>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<svg>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<use href=\"https:\/\/medx.b-cdn.net\/tmpl\/v6\/img\/svg\/sprite.svg#icon_open\" x=\"0\" y=\"0\"\/>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/svg><br \/>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/a><\/p><\/div>\n<p>                                        <!-- print only --><\/p>\n<div class=\"d-none d-print-block\">\n<p>\n                                                 <strong>Citation<\/strong>:<br \/>\n                                                 Gene therapy reduces stroke risk factors in sickle cell disease patients (2025, June 28)<br \/>\n                                                 retrieved 28 June 2025<br \/>\n                                                 from https:\/\/medicalxpress.com\/<a href=\"https:\/\/buradabiliyorum.com\/en\/category\/news\/\" data-internallinksmanager029f6b8e52c=\"2\" title=\"News\" target=\"_blank\" rel=\"noopener\">news<\/a>\/2025-06-gene-therapy-factors-sickle-cell.html\n                                            <\/p>\n<p>\n                                            This document is subject to copyright. 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Credit: St. Jude Children&#8217;s Research Hospital Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a&#8230;<\/p>\n","protected":false},"author":1,"featured_media":677698,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"fifu_image_url":"https:\/\/scx2.b-cdn.net\/gfx\/news\/hires\/2025\/gene-therapy-improves.jpg","fifu_image_alt":"","footnotes":""},"categories":[16],"tags":[],"class_list":["post-677697","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-sciencee"],"_links":{"self":[{"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/posts\/677697","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/comments?post=677697"}],"version-history":[{"count":0,"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/posts\/677697\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/media\/677698"}],"wp:attachment":[{"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/media?parent=677697"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/categories?post=677697"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/tags?post=677697"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}