{"id":7243,"date":"2018-10-04T11:59:47","date_gmt":"2018-10-04T11:59:47","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=7243"},"modified":"2022-05-03T18:14:48","modified_gmt":"2022-05-03T18:14:48","slug":"chimeric-cells-dmd","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/chimeric-cells-dmd\/","title":{"rendered":"&#8220;Chimeric cells\u201d for DMD?"},"content":{"rendered":"<h3>Cells Restoring dystrophin in DMD<\/h3>\n<p><strong>We\u2019ve set a goal for ourselves of informing you about new treatments for Duchenne muscular dystrophy (DMD). Here are some very promising results: new research conducted by Dr.\u00a0Maria Siemionow, professor of orthopedic surgery in the UIC College of Medicine. Good reading and thank you for sharing these articles within the DMD community.<\/strong><\/p>\n<p>&nbsp;<\/p>\n<h3>What is the role of dystrophin in Duchenne muscular dystrophy (DMD)?<\/h3>\n<p>Duchenne muscular dystrophy (DMD) is a degenerative muscle disease caused by a genetic mutation. Duchenne muscular dystrophy (DMD)\u2014for which no treatment is currently available\u2014directly affects the muscles. The simplest way to explain this disease is that a genetic mutation (i.e. a genetic defect) affects the gene responsible for the production of dystrophin. This genetic defect prevents the gene from producing dystrophin. In the absence of dystrophin, the muscles degenerate and become atrophied.<\/p>\n<p>&nbsp;<\/p>\n<h3>What are \u201cchimeric cells\u201d?<\/h3>\n<p>These cells, called \u201cchimeric cells,\u201d are made by combining a normal donor cell containing a functional copy of the dystrophin gene with a cell from a recipient with the disease. These cells were able to significantly improve muscle function when implanted into the muscles of a mouse model of the disease.<\/p>\n<p>&nbsp;<\/p>\n<h3>Research with promising results<\/h3>\n<p>Dr.\u00a0Maria Siemionow, professor of orthopedic surgery in the UIC College of Medicine, and her team used \u201cchimeric cells\u201d in a study of model mice with DMD. The results are excellent: the boosted dystrophin levels by 37 percent and improved muscle function when implanted into the muscles of a mouse model of Duchenne muscular dystrophy. These cells remained viable and produced dystrophin for 30 days.<\/p>\n<p>&nbsp;<\/p>\n<blockquote><p>\u201cOur results point to the long-term survival of these cells and helps establish the use of chimeric cells as a novel promising potential therapy for patients with Duchenne muscular dystrophy,\u201d Siemionow said.<\/p><\/blockquote>\n<p>&nbsp;<\/p>\n<h3>Clinical trials for on humans forthcoming<\/h3>\n<p>Dr.\u00a0Maria Siemionow also mentioned that her team is looking forward to clinical trials in humans shortly. She went on to say:<\/p>\n<p>&nbsp;<\/p>\n<blockquote><p>\u201cWe are restoring dystrophin in such a way that the recipient won\u2019t need to take anti-rejection therapy because the implanted chimeric cells can evade the recipient\u2019s immune system. In traditional stem cell therapy, the implanted cells are 100 percent \u2018other\u2019 and anti-rejection medicine is needed in order to prevent the host immune system from destroying those foreign cells.\u201d<\/p><\/blockquote>\n<p>&nbsp;<\/p>\n<h3>How do chimeric cells work on humans?<\/h3>\n<p>If these cells were to be used to treat a patient with Duchenne muscular dystrophy, then normal muscle cells from the father or a close relative of the recipient would be fused with muscle cells from the patient. In contrast, chimeric cells can trick the recipient\u2019s immune system into ignoring them. In lab tests, it was possible to bring the chimeric cells to express dystrophin.<\/p>\n<p>Maria and Kris Siemionow, who are mother and son, have recently launched a company to develop their chimeric cells into a therapy to treat Duchenne muscular dystrophy called Dystrogen Therapeutics. The development of such a therapy is captivating, and its evolution deserves to be followed.<\/p>\n<p>&nbsp;<\/p>\n<h3>External links and special thanks to:<\/h3>\n<p><a href=\"http:\/\/dystrogen.com\/\" rel=\"noopener\"><strong>Dystrogen.com:<\/strong>\u00a0<\/a><a href=\"http:\/\/dystrogen.com\/\" rel=\"noopener\">Clinical stage regenerative medicine company<\/a><\/p>\n<p><a href=\"https:\/\/scicasts.com\/channels\/genomics\/2015-gene-therapy\/13424-human-chimeric-cells-restore-crucial-protein-in-duchenne-muscular-dystrophy\/\" rel=\"noopener\"><strong>Scicasts.com:<\/strong>\u00a0Human \u201cChimeric\u201d Cells Restore Crucial Protein in Duchenne Muscular Dystrophy\u00a0<\/a><\/p>\n<p lang=\"en\"><a href=\"https:\/\/link.springer.com\/article\/10.1007%2Fs12015-018-9807-z\" rel=\"noopener\"><strong>Springer Link:<\/strong>\u00a0Dystrophin Expressing Chimeric (DEC) Human Cells Provide a Potential Therapy for Duchenne Muscular Dystrophy<\/a><\/p>\n<p><a href=\"https:\/\/medicine.uic.edu\/\" rel=\"noopener\"><strong>UIC College of Medicine<\/strong><\/a><\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Cells Restoring dystrophin in DMD We\u2019ve set a goal for ourselves of informing you about new treatments for Duchenne muscular dystrophy (DMD). Here are some very promising results: new research conducted by Dr.\u00a0Maria Siemionow, professor of orthopedic surgery in the UIC College of Medicine. Good reading and thank you for sharing these articles within the [&hellip;]<\/p>\n","protected":false},"author":2,"featured_media":7246,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[17],"tags":[],"class_list":["post-7243","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-research"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>&quot;Chimeric cells\u201d for DMD?<\/title>\n<meta name=\"description\" content=\"Here are some very promising results: new research conducted by Dr.\u00a0Maria Siemionow, about DMD and &quot;Chimeric cells\u201d, can restore dystrophin in DMD.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" 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