{"id":265356,"date":"2021-06-02T19:50:45","date_gmt":"2021-06-02T16:50:45","guid":{"rendered":"https:\/\/en.buradabiliyorum.com\/newer-methods-may-boost-gene-therapys-use-for-more-diseases-2\/"},"modified":"2021-06-02T19:50:45","modified_gmt":"2021-06-02T16:50:45","slug":"newer-methods-may-boost-gene-therapys-use-for-more-diseases-2","status":"publish","type":"post","link":"https:\/\/buradabiliyorum.com\/en\/newer-methods-may-boost-gene-therapys-use-for-more-diseases-2\/","title":{"rendered":"#Newer methods may boost gene therapy\u2019s use for more diseases"},"content":{"rendered":"<p>&#8220;<strong>#Newer methods may boost gene therapy\u2019s use for more diseases<\/strong>&#8221;<br \/>\n<img decoding=\"async\" src=\"https:\/\/nypost.com\/wp-content\/uploads\/sites\/2\/2021\/06\/gene-therapy.jpg?quality=90&amp;strip=all\" \/><\/p>\n<div>\n<p>Jordan Janz knew his gamble on an experimental gene therapy for his rare disease might be paying off when he returned to work and a friend sniffed him.<\/p>\n<p>\u201cHe said, \u2018you have a normal smell, you smell good,\u2019\u201d Janz recalled. \u201cAnd I\u2019m like, \u2018that\u2019s probably the nicest thing you\u2019ve ever said.\u2019\u201d<\/p>\n<p>The 22-year-old Canadian man\u2019s previous treatment required 40 to 60 pills a day and left him smelling like rotten eggs or stinky cheese. He was born with a flawed gene that left him unable to make a protein needed by virtually every organ in the body. Kids with this disease can throw up a dozen times a day, need eye drops every hour to prevent blindness and often kidney transplants before they\u2019re adults.<\/p>\n<p>Now, Janz and a growing number of others with rare diseases have hope of not just better treatments, but perhaps a cure. Gene therapy increasingly is being used to attack the root cause of their problems by supplying the DNA they lack.<\/p>\n<p>Janz was the first person in one such study at the University of California, San Diego. He and two other participants no longer need the eye drops and pills that only delayed progression of their disease and left them smelling bad. The company that developed their therapy is testing it for several other diseases by simply adjusting what gene is supplied.<\/p>\n<p>Other companies are doing the same and US health officials are working on guidance to encourage the trend.<\/p>\n<p>\u201cI am very excited for the field because I feel like we\u2019re beginning to get to a critical mass,\u201d where a single method or product can be deemed safe and then adapted for many uses, said Dr. Peter Marks, head of the US Food and Drug Administration center that oversees gene therapies. \u201cYou\u2019re essentially using the same rocket ship to put stuff into space dozens of times.\u201d<\/p>\n<p><strong>Not so rare diseases <\/strong><\/p>\n<figure class=\"wp-block-embed-youtube wp-block-embed is-type-video is-provider-youtube wp-embed-aspect-16-9 wp-has-aspect-ratio\">\n<span class=\"embed-youtube\" style=\"text-align:center; display: block;\"><iframe loading=\"lazy\" title=\"Increase in use of gene therapy for rare diseases\" width=\"640\" height=\"360\" src=\"https:\/\/www.youtube.com\/embed\/SHt7tX-SsHI?feature=oembed\" frameborder=\"0\" allow=\"accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share\" referrerpolicy=\"strict-origin-when-cross-origin\" allowfullscreen><\/iframe><\/span>\n<\/p>\n<\/figure>\n<p>In the United States, a disease is considered rare if it affects fewer than 200,000 people, said Ron Bartek, a board member of the\u00a0<a rel=\"nofollow noopener\" target=\"_blank\" href=\"https:\/\/rarediseases.org\/\" class=\"\">National Organization for Rare Disorders<\/a>\u00a0and whose son, Keith, died of one \u2014 Friedreich ataxia, a progressive neurological condition.<\/p>\n<p>There are more than 7,000 rare diseases and collectively, they\u2019re not so rare \u2014 about 30 million Americans have one.<\/p>\n<p>Seven gene therapies are <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>roved in the United States and a few more are sold in Europe or elsewhere. In 2017, when the first ones were OK\u2019d in the US, 854 companies were developing them. That grew to 1,085 by the end of last year, according to the Alliance for Regenerative Medicine, an advocacy group for the field. More than 400 gene therapy trials are underway now.<\/p>\n<p>\u201cIt\u2019s really been an exciting couple of years,\u201d Bartek said. \u201cWe\u2019re finally looking at the possibility of profoundly beneficial therapies\u201d for many diseases.<\/p>\n<p>There\u2019s interest from not only small biotech firms but also universities and big companies such as Pfizer and Novartis, he said.<\/p>\n<p><strong>Doctoring DNA<\/strong><\/p>\n<p>Gene therapies often aim to supply a gene people lack. The trick is getting the new one where it needs to be without triggering other problems.<\/p>\n<p>Some therapies, such as ones for inherited forms of blindness, inject the treatment into the eye. A common method for others is to remove some of a patient\u2019s blood cells, alter them in the lab to carry the desired gene and return them through an IV.<\/p>\n<p>What has scientists excited is better vectors \u2014 disabled viruses that ferry the gene into cells \u2014 that seem safer and more effective.<\/p>\n<p>One, surprisingly, may be HIV, the virus that causes AIDS. Doctors recently reported that a gene therapy using modified HIV from London-based Orchard Therapeutics gave a working immune system to 48 babies and toddlers who were born without one. Avrobio, a Massachusetts company, also is using HIV in gene therapies it is testing for cystinosis, Janz\u2019s disease and several others.<\/p>\n<p>BioMarin Pharmaceutical Inc. in California is taking a similar approach with a different virus called AAV for hemophilia, a blood clotting disorder and several other diseases.<\/p>\n<p>\u201cThe only thing that\u2019s changing is the payload, the gene,\u201d said Avrobio\u2019s chief scientific officer, Dr. Chris Mason.<\/p>\n<p><strong>The sniff test <\/strong><\/p>\n<p>Janz lives in Consort, a Canadian village of 700 in Alberta. His mother called the first day of enrollment for Avrobio\u2019s study under Stephanie Cherqui, a pediatrics expert at UCSD.<\/p>\n<p>People with cystinosis are unable to clear cystine, a chemical that builds up in their cells and forms crystals that damage kidneys, eyes and other organs and leads to muscle weakness, trouble swallowing and even breathing. The vomiting is so bad that many kids need a feeding tube placed in their stomach and wind up being home-schooled because they\u2019re teased by classmates and their medical needs are so complex, Cherqui said.<\/p>\n<p>\u201cWhen you meet these families, these children are suffering every day from the disease,,\u201d she said. \u201cI can\u2019t even imagine. It\u2019s terrible.\u201d<\/p>\n<p>Gene therapy offers hope of a one-time fix. The therapy doesn\u2019t have to get into all cells to work, just enough of them to make sufficient amounts of the protein that removes cystine so it doesn\u2019t build up.<\/p>\n<p>Janz was treated in 2019 and said he feels \u201cessentially cured,\u201d though it will take longer to know if he really is and he may need a kidney transplant someday because of damage done by his disease. Tests show that crystals in his eyes, skin and muscle have greatly decreased. Instead of 54 pills a day, he just takes vitamins and specific nutrients his body needs.<\/p>\n<p>\u201cI have more of a life now,\u201d Janz said. \u201cI\u2019m going to school. I\u2019m hoping to open up my own business one day\u201d \u2014 a deli featuring charcuterie, prepared meats such as hams and sausages. It\u2019s an interest he developed during years of frequent <a href=\"https:\/\/buradabiliyorum.com\/en\/category\/trip-and-travel\/\" data-internallinksmanager029f6b8e52c=\"10\" title=\"Trip &amp; Travel\" target=\"_blank\" rel=\"noopener\">trip<\/a>s to Chicago as a young boy to try other treatments. He stayed so often at the Omni Hotel that the chef let him cook in the hotel\u2019s fancy restaurant when he was 13. The staff even bought Janz a knife kit and his own chef\u2019s hat, apron and shoes.<\/p>\n<p><strong>Challenges remain <\/strong><\/p>\n<p>Gene therapy doesn\u2019t work for everyone, it\u2019s unknown how long any benefits will last and safety remains a concern. In the past, a couple gene therapies that used different vectors triggered cancer in a few patients who received them.<\/p>\n<p>\u201cEvery time we think we have overcome the safety issues, we continue to butt up against them,\u201d the FDA\u2019s Marks said. \u201cI have confidence that we will work through them \u2026 but we can\u2019t get too sure of ourselves.\u201d<\/p>\n<p>Marks and many other scientists think gene editing eventually may offer a long-lasting way to do gene therapy. Instead of just supplying a missing gene to cells, editing permanently alters a patient\u2019s native DNA to insert a gene or knock out one that\u2019s causing trouble.<\/p>\n<p>Brian Madeux, a Phoenix-area man who is now 48, became the\u00a0<a rel=\"nofollow noopener\" target=\"_blank\" href=\"https:\/\/apnews.com\/article\/genetics-gene-therapy-genetic-frontiers-california-oakland-4ae98919b52e43d8a8960e0e260feb0a?utm_campaign=socialflow&amp;utm_source=twitter&amp;utm_medium=ap\">first person in the world to have gene editing<\/a>\u00a0tried inside his body in 2017 for a metabolic disorder called Hunter syndrome. Through an IV, he received billions of copies of a corrective gene and a genetic tool to put it in a precise spot.<\/p>\n<p>But the therapy wasn\u2019t potent enough to allow him or eight other patients to stop weekly IV treatments to supply the enzyme their bodies are unable to make.<\/p>\n<p>Even though it didn\u2019t give the hoped-for result, \u201cI think the doctors have learned a lot from me,\u201d Madeux said recently. \u201cI felt that I could help other people\u201d by furthering <a href=\"https:\/\/buradabiliyorum.com\/en\/category\/sciencee\/\" data-internallinksmanager029f6b8e52c=\"5\" title=\"Science\" target=\"_blank\" rel=\"noopener\">science<\/a>.<\/p>\n<p>\u201cI took a shot, a chance that something could cure my disease,\u201d he said. \u201cI\u2019m very happy that I did it.\u201d\n            <\/p><\/div>\n<blockquote><p><strong><span style=\"color: #ff6600;\">If you liked the article, do not forget to share it with your friends. Follow us on\u00a0<span style=\"color: #ff0000;\"><a style=\"color: #ff0000;\" href=\"https:\/\/news.google.com\/publications\/CAAqBwgKMLG0nwswvr63Aw\" target=\"_blank\" rel=\"nofollow noopener noreferrer\">Google News<\/a><\/span>\u00a0too, click on the star and choose us from your favorites.<\/span><\/strong><\/p><\/blockquote>\n<blockquote>\n<p style=\"text-align: center;\">For forums sites go to <span style=\"color: #ff9900;\"><a style=\"color: #ff9900;\" href=\"https:\/\/forum.buradabiliyorum.com\/\" target=\"_blank\" rel=\"noopener\">Forum.BuradaBiliyorum.Com<\/a><\/span><\/strong>\n<\/p><\/blockquote>\n<blockquote>\n<p style=\"text-align: center;\"><strong>If you want to read more <a href=\"https:\/\/buradabiliyorum.com\/en\/category\/news\/\" data-internallinksmanager029f6b8e52c=\"2\" title=\"News\" target=\"_blank\" rel=\"noopener\">News<\/a> articles, you can visit our <span style=\"color: #ff9900;\"><a style=\"color: #ff9900;\" href=\"https:\/\/en.buradabiliyorum.com\/news\/\" target=\"_blank\" rel=\"noopener\">News category.<\/a><\/span><\/strong><\/p>\n<\/blockquote>\n<p><span style=\"color: black;\"><a style=\"color: #ff9900;\" href=\"https:\/\/nypost.com\/2021\/06\/02\/newer-methods-may-boost-gene-therapys-use-for-more-diseases\/\" target=\"_blank\" rel=\"noopener\">Source<\/a><\/span><\/p>\n","protected":false},"excerpt":{"rendered":"<p>&#8220;#Newer methods may boost gene therapy\u2019s use for more diseases&#8221; Jordan Janz knew his gamble on an experimental gene therapy for his rare disease might be paying off when he returned to work and a friend sniffed him. \u201cHe said, \u2018you have a normal smell, you smell good,\u2019\u201d Janz recalled. \u201cAnd I\u2019m like, \u2018that\u2019s probably&#8230;<\/p>\n","protected":false},"author":1,"featured_media":265357,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"fifu_image_url":"https:\/\/nypost.com\/wp-content\/uploads\/sites\/2\/2021\/06\/gene-therapy.jpg?quality=90&strip=all&w=1200","fifu_image_alt":"","footnotes":""},"categories":[70897],"tags":[108053,73461,70510,61514,5050,4965],"class_list":["post-265356","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","tag-6-2-21","tag-diseases","tag-genetics","tag-research","tag-scientists","tag-technology"],"_links":{"self":[{"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/posts\/265356","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=265356"}],"version-history":[{"count":0,"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/posts\/265356\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/media\/265357"}],"wp:attachment":[{"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/media?parent=265356"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/categories?post=265356"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/buradabiliyorum.com\/en\/wp-json\/wp\/v2\/tags?post=265356"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}