{"id":23673,"date":"2025-03-10T20:06:15","date_gmt":"2025-03-10T21:06:15","guid":{"rendered":"http:\/\/medexperts.pro\/?p=23673"},"modified":"2025-03-10T21:25:54","modified_gmt":"2025-03-10T21:25:54","slug":"mutated-dna-restored-to-normal-in-gene-therapy-advance","status":"publish","type":"post","link":"https:\/\/medexperts.pro\/?p=23673","title":{"rendered":"Mutated DNA Restored to Normal in Gene Therapy Advance"},"content":{"rendered":"
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The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely targeted infusions.<\/p>\n

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Researchers have corrected a disease-causing gene mutation with a single infusion carrying a treatment that precisely targeted the errant gene.<\/p>\n

This was the first time a mutated gene has been restored to normal.<\/p>\n

The small study<\/a> of nine patients announced Monday by the company Beam Therapeutics of Cambridge, Mass., involved fixing a spelling error involving the four base sequences \u2014 G, A, C and T \u2014 in DNA. The effect was to change an incorrect DNA letter to the right one. The result was a normal gene that functioned as it should, potentially halting liver and lung damage of patients with a rare disorder.<\/p>\n

\u201cThis is the beginning of a new era of medicine,\u201d said Dr. Kiran Musunuru, a gene therapy researcher at the University of Pennsylvania\u2019s Perelman School of Medicine.<\/p>\n

He added that the method offers the hope of treating other genetic diseases precisely by fixing mutations \u2014 an alternative to current gene therapies, which either add new genes to compensate for mutated ones, or slicing DNA to silence genes.<\/p>\n<\/div>\n<\/div>\n

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Dr. Musunuru is a co-founder and equity holder of Verve Therapeutics, a gene therapy company, and receives funding from Beam Therapeutics for research, but not for this study.<\/p>\n

Dr. Richard P. Lifton, president of Rockefeller University and head of its Laboratory of Human Genetics and Genomics, said the sort of gene editing Beam did, rewriting genes with an infusion, \u201cis a holy grail\u201d that \u201chas the promise for being a one-and-done kind of therapy.\u201d<\/p>\n

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The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely targeted infusions.Researchers have corrected a disease-causing gene mutation with a single infusion carrying a treatment that precisely targeted the errant gene.This was the first time a mutated gene has been restored to normal.The small study of nine patients announced Monday by the company Beam Therapeutics of Cambridge, Mass., involved fixing a spelling error involving the four base sequences \u2014 G, A, C and T \u2014 in DNA. The effect was to change an incorrect DNA letter to the right one. The result was a normal gene that functioned as it should, potentially halting liver and lung damage of patients with a rare disorder.\u201cThis is the beginning of a new era of medicine,\u201d said Dr. Kiran Musunuru, a gene therapy researcher at the University of Pennsylvania\u2019s Perelman School of Medicine.He added that the method offers the hope of treating other genetic diseases precisely by fixing mutations \u2014 an alternative to current gene therapies, which either add new genes to compensate for mutated ones, or slicing DNA to silence genes.Dr. Musunuru is a co-founder and equity holder of Verve Therapeutics, a gene therapy company, and receives funding from Beam Therapeutics for research, but not for this study.Dr. Richard P. Lifton, president of Rockefeller University and head of its Laboratory of Human Genetics and Genomics, said the sort of gene editing Beam did, rewriting genes with an infusion, \u201cis a holy grail\u201d that \u201chas the promise for being a one-and-done kind of therapy.\u201dWe are having trouble retrieving the article content.Please enable JavaScript in your browser settings.Thank you for your patience while we verify access. If you are in Reader mode please exit and\u00a0log into\u00a0your Times account, or\u00a0subscribe\u00a0for all of The Times.Thank you for your patience while we verify access.Already a subscriber?\u00a0Log in.Want all of The Times?\u00a0Subscribe.<\/p>\n","protected":false},"author":1,"featured_media":23675,"comment_status":"close","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[34],"tags":[],"class_list":["post-23673","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-science"],"_links":{"self":[{"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/posts\/23673","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/medexperts.pro\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=23673"}],"version-history":[{"count":2,"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/posts\/23673\/revisions"}],"predecessor-version":[{"id":23676,"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/posts\/23673\/revisions\/23676"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/medexperts.pro\/index.php?rest_route=\/wp\/v2\/media\/23675"}],"wp:attachment":[{"href":"https:\/\/medexperts.pro\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=23673"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/medexperts.pro\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=23673"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/medexperts.pro\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=23673"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}