developed<\/a> by UC Berkeley researchers Jennifer Doudna and Emmanuelle Charpentier in 2012.\u00a0<\/p>\nIn only six months, the researchers developed a personalized treatment to combat the effects of Muldoon\u2019s genetic disorder.<\/p>\n
\u201cThat was really record-breaking time,\u201d said principal investigator at IGI Petros Giannikopoulos.<\/p>\n
The treatment utilized CRISPR technology to \u201cedit\u201d the patient\u2019s liver cells into producing the enzyme CPS-1, which breaks down ammonia by incorporating it into urea, according to Giannikopoulos. This was done by using a lipid nanoparticle and a CRISPR-modified RNA molecule to target the specific site of the disorder, \u201cediting\u201d the cells there to produce CPS-1.<\/p>\n
Muldoon received his first of three doses in February. After the first doses were administered, the researchers used Muldoon\u2019s dietary responses to measure his progress.<\/p>\n
The researchers found that Muldoon\u2019s blood urea nitrogen levels were reduced following the start of the treatment, indicating he was responding well.\u00a0<\/p>\n
Doctors and dietitians have continued to monitor Muldoon, noting through his diet and general wellness checks that he is growing up healthily.<\/p>\n
The IGI research team led by Giannikopoulos, which included campus Ph.D. student Taylor Hudson, campus undergraduate Aysel Ogul and research associate Kevin Briseno, was responsible for evaluating the potential risks of the process and ensuring its safety for the patient.<\/p>\n
\u201cA lot of these cases, including this, (involve) such rare diseases that no pharmaceutical company, no big industry is going to make a treatment for them,\u201d Hudson said while explaining why a specialized treatment is necessary for cases like Muldoon\u2019s.<\/p>\n
While this is not the first time CRISPR has been used to treat a patient,Muldoon\u2019s treatment is revolutionary because it is the first time CRISPR therapy has been designed specifically for an individual, according to Giannikopoulos.\u00a0<\/p>\n
Researchers hope to apply this process on a larger scale in the future, using early cases such as Muldoon\u2019s as a \u201cblueprint,\u201d Giannikopoulos added.<\/p>\n
\u201cThis was a reactive process, not a proactive process,\u201d Giannikopoulos said. \u201cWe reacted to this patient\u2019s specific mutation, and that was what was so groundbreaking.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"Researchers at UC Berkeley\u2019s Innovative Genomics Institute, or IGI, developed a revolutionary treatment using CRISPR-Cas9 gene editing technology…\n","protected":false},"author":2,"featured_media":117047,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3846],"tags":[52535,52530,14192,52532,267,52528,52531,52536,52529,52533,70,52534,16,15],"class_list":{"0":"post-117046","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-genetics","8":"tag-aysel-ogul","9":"tag-childrens-hospital-of-philadelphia","10":"tag-crispr","11":"tag-emmanuelle-charpentier","12":"tag-genetics","13":"tag-innovative-genomics-institute","14":"tag-jennifer-doudna","15":"tag-kevin-briseno","16":"tag-kj-muldoon","17":"tag-petros-giannikopoulos","18":"tag-science","19":"tag-taylor-hudson","20":"tag-uk","21":"tag-united-kingdom"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@uk\/114540157118693934","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/117046","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/comments?post=117046"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/117046\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/117047"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=117046"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=117046"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=117046"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}