{"id":396609,"date":"2025-09-04T07:00:19","date_gmt":"2025-09-04T07:00:19","guid":{"rendered":"https:\/\/www.europesays.com\/uk\/396609\/"},"modified":"2025-09-04T07:00:19","modified_gmt":"2025-09-04T07:00:19","slug":"dream-team-to-develop-in-utero-gene-editing-therapy-for-a-rare-neurological-genetic-disorder-2","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/uk\/396609\/","title":{"rendered":"Dream team to develop in utero gene editing therapy for a rare neurological genetic disorder"},"content":{"rendered":"

(SACRAMENTO) <\/strong><\/p>\n

Scientists from\u00a0UC Davis Center for Surgical Bioengineering<\/a>, the\u00a0MIND Institute<\/a>\u00a0and UC Berkeley\u2019s\u00a0Murthy Lab<\/a>\u00a0are developing an in-utero gene editing technology to fix mutations causing severe neurologic conditions.<\/p>\n

Led by UC Davis Bioengineer\u00a0Aijun Wang<\/a>, the team will design and test a therapy targeting the UBE3A gene. Overexpression of this gene contributes to Dup15q syndrome, and its absence leads to Angelman syndrome. Both conditions are profound neurodevelopmental conditions that could co-occur with autism.<\/p>\n

Finding a therapy for Dup15q syndrome<\/p>\n

Dup15q syndrome<\/a>\u00a0affects about 1 in 5,000 individuals. It is 10 times more common in people with autism and intellectual disability. More than half of patients with Dup15q experience epilepsy.<\/p>\n

\u201cThere is no readily available cure for Dup15q syndrome. Caring for chronically ill children with this condition places a significant emotional and financial burden on their families,\u201d said Wang, professor of surgery and biomedical engineering. \u201cThe goal of the gene editing therapy we are developing is to one day treat Dup15q in utero so that children are born without the condition.\u201d<\/p>\n

Wang is the vice chair for translational research, innovation and entrepreneurship at the Department of Surgery. He co-directs the Center for Surgical Bioengineering and leads\u00a0Wang Lab<\/a>, a prime research hub in stem cell therapy and gene editing for early treatments of conditions such as\u00a0spina bifida<\/a>\u00a0and\u00a0Duchenne muscular dystrophy<\/a>.<\/p>\n

This new work will be funded by a 5-year $3.2 million grant from the National Institutes of Health\u2019s\u00a0National Institute of Neurological Disorders and Stroke.<\/a><\/p>\n

\"Dr.
\nWang Lab is a prime research hub in stem cell therapy and gene editing<\/p>\n

Early diagnosis allows for early intervention<\/p>\n

UC Davis fetal surgeon Diana Farmer<\/a>\u00a0and Wang\u00a0pioneered a groundbreaking spina bifida treatment<\/a>\u00a0by operating on developing babies in the womb.<\/p>\n

Now, they are expanding to nanomedicine to help find treatment to babies with genetic diseases. These diseases can cause progressive loss of function, so early intervention is critical.<\/p>\n

\u201cWe really have learned so much from our work on spina bifida about the fetal environment,\u201d Wang explained. \u201cIntervening early in gestation can change the disease escalation and prevent the severe consequences of the genetic mutation.\u201d<\/p>\n

Innovation in the delivery of the gene editing therapy<\/p>\n

This new therapy targets neural stem and progenitor cells during a critical time in fetal development. It is an injection that uses a lipid nanoparticle (LNP) to carry Cas9 mRNA \u2014 the code to make the gene-editing enzyme. This enzyme is supposed to genetically modify the stem cells and correct the mutation. When these cells multiply and migrate to form neurons in the brain, they would carry the corrected genes.<\/p>\n

Unlike delivery methods that use viruses to carry the gene editing therapy, this approach relies on LNPs. Wang\u2019s lab has developed the LNP technology in collaboration with UC Berkeley professor\u00a0Niren Murthy<\/a>. They screened many LNP formulations until they identified the ones that minimize toxicity and optimize efficiency when introduced to a developing fetus.<\/p>\n

\u201cA highly innovative aspect of this work is the nonviral delivery of the therapy,\u201d said\u00a0Jill Silverman<\/a>, professor in the\u00a0Department of Psychiatry and Behavioral Science<\/a>\u00a0and the MIND Institute. \u201cUsing viruses as therapy carriers has been an issue in developing and optimizing gene therapies for trials. It sometimes can cause intense immune reactions that could prevent future use of this virus.\u201d<\/p>\n

\n \"AijunGene editing is so powerful. A major barrier in the field of genetic diseases has been the absence of effective treatments for many of these conditions. But that is changing with the emerging gene editing and CRISPR technologies,\u201d Aijun Wang, UC Davis professor of bioengineering and surgery.\u2014Aijun Wang, UC Davis professor of surgery and biomedical engineering<\/p><\/blockquote>\n

Disease mouse models and brains in a dish<\/p>\n

The team has an ambitious three-pillar plan. They will:<\/p>\n

    \n
  1. Identify an LNP formulation that can be safely and efficiently used in utero. They will test the toxicity and efficiency of their formulation in delivering gene editing proteins to the neural stem and progenitor cells.<\/li>\n
  2. Observe the effect of this formulation in brain neurons of a Dup15q mouse model that overexpresses the\u00a0Ube3a\u00a0Silverman<\/a>\u00a0has identified relevant outcome measures and biomarkers of the disease that will be studied in the treated Dup15q mice.<\/li>\n
  3. Test the formulation in a brain-in-a-dish model. This approach uses human stem cells that grow into brain organoids in a petri dish to mimic working human brain neurons. These organoids provide researchers the opportunity to assess the effects and compare them to those found in the mouse model.<\/li>\n<\/ol>\n

    \"Dr.
    \nProfessors Aijun Wang and Diana Farmer with members of their labs at UC Davis<\/p>\n

    Team science approach to tackle medicine\u2019s toughest genetic conditions<\/p>\n

    This study is co-led by Wang, Murthy and Silverman. It also brings together top-notch experts at UC Davis, including:<\/p>\n

    Wang and his team are planning to target different severe and early diagnosable genetic problems that can be fixed by gene editing. They are also exploring using this core technology platform to help patients with fetal conditions, such as Duchenne muscular dystrophy.<\/p>\n

    \u201cGene editing is so powerful. A major barrier in the field of genetic diseases has been the absence of effective treatments. But that is changing with the emerging gene editing and\u00a0CRISPR technologies<\/a>. Hopefully soon, many more diseases will be screened for during gestation, and cures will be available before birth,\u201d Wang said.<\/p>\n

    Related Stories:<\/p>\n

    World\u2019s first stem cell treatment for spina bifida delivered during fetal surgery<\/strong><\/a><\/p>\n

    UC Davis Health to develop in-utero therapy for Duchenne muscular dystrophy<\/strong><\/a><\/p>\n","protected":false},"excerpt":{"rendered":"(SACRAMENTO) Scientists from\u00a0UC Davis Center for Surgical Bioengineering, the\u00a0MIND Institute\u00a0and UC Berkeley\u2019s\u00a0Murthy Lab\u00a0are developing an in-utero gene editing…\n","protected":false},"author":2,"featured_media":395773,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3846],"tags":[267,70,16,15],"class_list":{"0":"post-396609","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-genetics","8":"tag-genetics","9":"tag-science","10":"tag-uk","11":"tag-united-kingdom"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@uk\/115144741904202625","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/396609","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=396609"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/396609\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/395773"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=396609"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=396609"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=396609"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}