{"id":24669,"date":"2025-05-21T15:52:24","date_gmt":"2025-05-21T15:52:24","guid":{"rendered":"https:\/\/www.tun.com\/home\/?p=24669"},"modified":"2025-05-21T15:54:02","modified_gmt":"2025-05-21T15:54:02","slug":"breakthrough-als-research-opens-path-for-early-treatment","status":"publish","type":"post","link":"https:\/\/www.tun.com\/home\/breakthrough-als-research-opens-path-for-early-treatment\/","title":{"rendered":"Breakthrough ALS Research Opens Path for Early Treatment"},"content":{"rendered":"\n<div class=\"wp-block-group\"><div class=\"wp-block-group__inner-container is-layout-constrained wp-block-group-is-layout-constrained\">\n<div class=\"wp-block-uagb-blockquote uagb-block-e7eb3fc3 uagb-blockquote__skin-border uagb-blockquote__stack-img-none\"><blockquote class=\"uagb-blockquote\"><div class=\"uagb-blockquote__content\">A new study by Stockholm University and King\u2019s College London uncovers early mitochondrial dysfunction in ALS, offering new hope for early intervention and treatment.<\/div><footer><div class=\"uagb-blockquote__author-wrap uagb-blockquote__author-at-left\"><\/div><\/footer><\/blockquote><\/div>\n\n\n\n<div class=\"wp-block-group is-content-justification-space-between is-nowrap is-layout-flex wp-container-core-group-is-layout-b0ffac9c wp-block-group-is-layout-flex\"><div style=\"font-size:16px\" class=\"has-text-align-left wp-block-post-author\"><div class=\"wp-block-post-author__content\"><p class=\"wp-block-post-author__name\">The University Network<\/p><\/div><\/div>\n\n\n<div class=\"wp-block-uagb-social-share uagb-social-share__outer-wrap uagb-social-share__layout-horizontal uagb-block-ee584a31\">\n<div class=\"wp-block-uagb-social-share-child uagb-ss-repeater uagb-ss__wrapper uagb-block-ec619ce7\"><span class=\"uagb-ss__link\" data-href=\"https:\/\/www.facebook.com\/sharer.php?u=\" tabindex=\"0\" role=\"button\" aria-label=\"facebook\"><span class=\"uagb-ss__source-wrap\"><span class=\"uagb-ss__source-icon\"><svg xmlns=\"https:\/\/www.w3.org\/2000\/svg\" viewBox=\"0 0 512 512\"><path d=\"M504 256C504 119 393 8 256 8S8 119 8 256c0 123.8 90.69 226.4 209.3 245V327.7h-63V256h63v-54.64c0-62.15 37-96.48 93.67-96.48 27.14 0 55.52 4.84 55.52 4.84v61h-31.28c-30.8 0-40.41 19.12-40.41 38.73V256h68.78l-11 71.69h-57.78V501C413.3 482.4 504 379.8 504 256z\"><\/path><\/svg><\/span><\/span><\/span><\/div>\n\n\n\n<div class=\"wp-block-uagb-social-share-child uagb-ss-repeater uagb-ss__wrapper uagb-block-32d99934\"><span class=\"uagb-ss__link\" data-href=\"https:\/\/twitter.com\/share?url=\" tabindex=\"0\" role=\"button\" aria-label=\"twitter\"><span class=\"uagb-ss__source-wrap\"><span class=\"uagb-ss__source-icon\"><svg xmlns=\"https:\/\/www.w3.org\/2000\/svg\" viewBox=\"0 0 512 512\"><path d=\"M389.2 48h70.6L305.6 224.2 487 464H345L233.7 318.6 106.5 464H35.8L200.7 275.5 26.8 48H172.4L272.9 180.9 389.2 48zM364.4 421.8h39.1L151.1 88h-42L364.4 421.8z\"><\/path><\/svg><\/span><\/span><\/span><\/div>\n\n\n\n<div class=\"wp-block-uagb-social-share-child uagb-ss-repeater uagb-ss__wrapper uagb-block-1d136f14\"><span class=\"uagb-ss__link\" data-href=\"https:\/\/www.linkedin.com\/shareArticle?url=\" tabindex=\"0\" role=\"button\" aria-label=\"linkedin\"><span class=\"uagb-ss__source-wrap\"><span class=\"uagb-ss__source-icon\"><svg xmlns=\"https:\/\/www.w3.org\/2000\/svg\" viewBox=\"0 0 448 512\"><path d=\"M416 32H31.9C14.3 32 0 46.5 0 64.3v383.4C0 465.5 14.3 480 31.9 480H416c17.6 0 32-14.5 32-32.3V64.3c0-17.8-14.4-32.3-32-32.3zM135.4 416H69V202.2h66.5V416zm-33.2-243c-21.3 0-38.5-17.3-38.5-38.5S80.9 96 102.2 96c21.2 0 38.5 17.3 38.5 38.5 0 21.3-17.2 38.5-38.5 38.5zm282.1 243h-66.4V312c0-24.8-.5-56.7-34.5-56.7-34.6 0-39.9 27-39.9 54.9V416h-66.4V202.2h63.7v29.2h.9c8.9-16.8 30.6-34.5 62.9-34.5 67.2 0 79.7 44.3 79.7 101.9V416z\"><\/path><\/svg><\/span><\/span><\/span><\/div>\n<\/div>\n<\/div>\n<\/div><\/div>\n\n\n\n<p class=\"wp-block-paragraph\">Researchers from Stockholm University and the UK Dementia Research Institute at King\u2019s College London have made a groundbreaking discovery in the study of Amyotrophic Lateral Sclerosis (ALS). Utilizing the advanced gene-editing tool CRISPR and stem cells, the team identified common mitochondrial dysfunctions in nerve cells affected by various ALS-related gene mutations, a revelation that could transform treatment approaches for this debilitating neurological disease.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Eva Hedlund, a professor of neurochemistry at Stockholm University, and her team discovered that mitochondrial issues in motor neurons appear long before other signs of the disease manifest.&nbsp;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cWe show that the nerve cells, termed motor neurons, that will eventually die in ALS have problems soon after they are formed. We saw the earliest sign of problems in the cell\u2019s energy factories, the mitochondria, and also in how they are transported out into the nerve cells\u2019 long processes where there is a great need for them and the energy they produce,\u201d Hedlund, who led the research alongside Marc-David Ruepp of the UK Dementia Research Institute at King\u2019s College London, said in a news release.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The findings,&nbsp;<a href=\"https:\/\/www.nature.com\/articles\/s41467-025-59679-1\">published<\/a>&nbsp;in the journal Nature Communications, mark a significant shift in understanding ALS\u2019s progression.&nbsp;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Historically, it was believed that mislocalization of proteins within cells was the initial step in disease development. However, this new study points to mitochondrial dysfunction as an early indicator, common across different gene mutations causing ALS. <\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The implications are profound, suggesting new avenues for drug targeting.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cThis means that there are common factors that could be targeted with drugs, regardless of the cause of the disease,\u201d Hedlund added.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The research demonstrated that whether mutated proteins were correctly or incorrectly located within cells, mitochondrial dysfunction persisted. This discovery challenges previous notions that protein mislocalization was the primary early event in ALS pathology.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cUntil now, it has been believed that it is the change where the proteins are within the cells, called mislocalization, that occurs first,\u201d Ruepp said in the news release.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">To reach these conclusions, the researchers employed CRISPR\/Cas9 to introduce ALS-causing mutations into human induced pluripotent stem cells (iPSCs). These cells were then differentiated into motor neurons and interneurons. Using single-cell RNA sequencing, they were able to identify a distinct disease signature specific to motor neurons affected by ALS mutations.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cIn the data we obtained, we identified a common disease signature across all ALS-causing mutations, which was unique to motor neurons and thus did not arise in resistant neurons,\u201d added first author Christoph Schweingruber, a research fellow at King\u2019s College London.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The team\u2019s work reveals potential strategies for early intervention. As Hedlund explains, understanding the early failure points in motor neurons and their impacts on cellular energy and communication could guide the development of treatments aimed at preserving neuron-muscle communication and preventing neuron death.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cBy making various CRISPR mutations in the ALS-causing FUS-gene, we have now been able to show for the first time that most errors arising are caused by a new toxic property of the protein, not by a loss of function,\u201d Schweingruber added.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The research highlights the critical role of mitochondria and their transport within nerve cells, particularly to axons, where energy demands are highest. Disruption in this transport mechanism could underlie the synaptic failures observed in ALS patients.&nbsp;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cWithout them, the nerve cells do not have enough energy to communicate properly with other cells,\u201d Hedlund added.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">In summary, these pivotal discoveries not only deepen our understanding of ALS but also open new pathways for therapeutic development. With ongoing research, the team aims to further demystify the disease\u2019s early stages and identify targets for novel treatments.<\/p>\n\n\n\n<div style=\"height:11px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Source:<\/strong> <a href=\"https:\/\/www.su.se\/english\/news\/new-research-on-als-opens-up-for-early-treatment-1.822423\" target=\"_blank\" rel=\"noopener\" title=\"\">Stockholm University<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Researchers from Stockholm University and the UK Dementia Research Institute at King\u2019s College London have made a groundbreaking discovery in the study of Amyotrophic Lateral Sclerosis (ALS). Utilizing the advanced gene-editing tool CRISPR and stem cells, the team identified common mitochondrial dysfunctions in nerve cells affected by various ALS-related gene mutations, a revelation that could [&hellip;]<\/p>\n","protected":false},"author":3,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"single-no-separators","format":"standard","meta":{"_acf_changed":false,"_uag_custom_page_level_css":"","_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0,"footnotes":""},"categories":[12],"tags":[50,49],"class_list":["post-24669","post","type-post","status-publish","format-standard","hentry","category-health","tag-kings-college-london","tag-stockholm-university"],"acf":[],"aioseo_notices":[],"uagb_featured_image_src":{"full":false,"thumbnail":false,"medium":false,"medium_large":false,"large":false,"1536x1536":false,"2048x2048":false},"uagb_author_info":{"display_name":"The University Network","author_link":"https:\/\/www.tun.com\/home\/author\/funky_junkie\/"},"uagb_comment_info":0,"uagb_excerpt":"Researchers from Stockholm University and the UK Dementia Research Institute at King\u2019s College London have made a groundbreaking discovery in the study of Amyotrophic Lateral Sclerosis (ALS). Utilizing the advanced gene-editing tool CRISPR and stem cells, the team identified common mitochondrial dysfunctions in nerve cells affected by various ALS-related gene mutations, a revelation that could&hellip;","_links":{"self":[{"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/posts\/24669","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/comments?post=24669"}],"version-history":[{"count":11,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/posts\/24669\/revisions"}],"predecessor-version":[{"id":24695,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/posts\/24669\/revisions\/24695"}],"wp:attachment":[{"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/media?parent=24669"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/categories?post=24669"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.tun.com\/home\/wp-json\/wp\/v2\/tags?post=24669"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}