{"id":260499,"date":"2025-09-28T05:08:11","date_gmt":"2025-09-28T05:08:11","guid":{"rendered":"https:\/\/www.europesays.com\/us\/260499\/"},"modified":"2025-09-28T05:08:11","modified_gmt":"2025-09-28T05:08:11","slug":"snuh-researchers-develop-worlds-first-injective-cure-for-congenital-deafness","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/us\/260499\/","title":{"rendered":"SNUH researchers develop world’s first injective cure for congenital deafness"},"content":{"rendered":"

\"Lee <\/p>\n

Lee Sang-yeon, a professor of pediatric otorhinolaryngology at Seoul National University Hospital (SNUH), speaks to a patient at SNUH in Jongno District, central Seoul on Sept. 19. [JUN MIN-KYU]<\/p>\n

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Korean researchers have developed the world\u2019s first gene-editing injection that can correct mutations causing hereditary deafness, offering new hope for children who have relied only on hearing aids or cochlear implants.
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\u201cMom, if I do this, can I get better too?\u201d
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Several months ago, nine-year-old Seo-jun (alias), who has lived with congenital hearing loss and relied on hearing aids all his life, asked his mother this question while undergoing a biopsy. At the time, no one could say whether treatment would be possible.
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Seo-jun\u2019s mother, who was heartbroken at not being able to give a definitive answer to her son\u2019s question, said in an interview with the JoongAng Ilbo on Wednesday, \u201cNow there is hope.\u201d
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Congenital deafness, like Seo-jun\u2019s, occurs in about one to three out of every 1,000 newborns. More than 200 different genes can cause it, and there has been no breakthrough in treatment beyond prescribing hearing aids or cochlear implants \u2014 until now.
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Lee Sang-yeon, a professor of pediatric otorhinolaryngology at Seoul National University Hospital and researcher in the Molecular Genome Engineering Laboratory led by Bae Sang-su, a professor of biochemistry at Seoul National University, recently reported that they have developed a gene-editing tool that produces meaningful hearing recovery.
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\"Lee <\/p>\n

Lee Sang-yeon, a professor of pediatric otorhinolaryngology at Seoul National University Hospital (SNUH), speaks during an interview with the JoongAng Ilbo at SNUH in Jongno District, central Seoul on Sept. 16. [JUN MIN-KYU]<\/p>\n

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The team targeted the MPZL2 mutation, observed in about 10 percent of hereditary deafness patients and found frequently in East Asians. This mutation often leads to rapid hearing decline during adolescence.
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The research team analyzed 1,437 families from a hereditary hearing loss cohort to identify the primary causative mutation. They then injected a mouse model with the newly developed gene editing agent. Hearing improved by 20 to 30 decibels, and the effect lasted more than five months.
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\u201cFor decades, the only treatments were hearing aids and cochlear implants,\u201d said Lee. \u201cNow gene therapy offers a new paradigm. The fact that hearing can be restored with a single injection, without surgery, is very encouraging.\u201d
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\u201cUntil now, all we could do was hope his hearing wouldn\u2019t get worse while he wore hearing aids,\u201d Seo-jun\u2019s mother said. \u201cHearing about this treatment gave me hope for the first time that my child may actually recover.\u201d
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\"A <\/p>\n

A diagram showing an overview of gene therapy study developed by a research team at Seoul National University Hospital for hearing loss. A single injection of the proprietary gene-editing tool resulted in improved hearing in a mouse model harboring the MPZL2 mutation, a major cause of hereditary sensorineural hearing loss. [SCREEN CAPTURE]<\/p>\n

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Mapping Korean deafness genes<\/b>
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The breakthrough was possible because the team built a \u201cKorean genetic map of deafness.\u201d Existing genetic testing could identify the cause in only about half of congenitally deaf patients. By conducting whole-genome sequencing on 752 patients and families, the team identified an additional 20 percent.
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They also discovered previously unknown mutations, creating opportunities for customized therapies.
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For instance, siblings Eun-chan (12, alias) and Eun-jin (9, alias), who have Usher syndrome, a condition that causes both hearing and vision loss with age, were found to carry a \u201cdeep intron mutation\u201d that previous tests could not detect. That discovery makes it possible to design a targeted treatment.
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\u201cThe MPZL2 therapy can be used for thousands of patients, but for rare intron mutations, \u2018N-of-1\u2019 treatments \u2014 designed for a single patient \u2014 are also necessary,\u201d explained Lee. \u201cDevelopment is essentially complete, but toxicity testing remains the key hurdle.\u201d
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\u201cI was terrified my kids would truly lose both their hearing and sight,\u201d said Eun-chan and Eun-jin\u2019s mother. \u201cHearing about this treatment made the future look brighter. It feels like a miracle, [something] beyond words.\u201d
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\"Lee <\/p>\n

Lee Sang-yeon, a professor of pediatric otorhinolaryngology at Seoul National University Hospital (SNUH), speaks during an interview with the JoongAng Ilbo at SNUH in Jongno District, central Seoul on Sept. 16. [JUN MIN-KYU]<\/p>\n

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Commercialization still faces challenges<\/b>
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Experts caution that there remains much to do before such treatments can become widely available. Safety testing requires significant time and funding, especially in medium-sized animals like monkeys.
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\u201cIt is important to treat hearing loss before it becomes too severe, so speed matters,\u201d said Lee. \u201cIn the United States, rare disease therapies can be fast-tracked. I hope Korea\u2019s drug regulators will take a flexible approach.\u201d
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The project was partly funded by the 300 billion won ($212.2 million) donation from the family of the late Samsung chairman Lee Kun-hee for childhood cancer and rare disease research, as well as support from the National Research Foundation of Korea and Seoul National University College of Medicine.
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\u201cBecause of research funding, we were able to assemble a strong team and make our ideas a reality,\u201d said Lee. \u201cI hope state support continues so this therapy can be safely developed and truly help patients.\u201d
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This article was originally written in Korean and translated by a bilingual reporter with the help of generative AI tools. It was then edited by a native English-speaking editor. All AI-assisted translations are reviewed and refined by our newsroom.
BY NAM SOO-HYOUN [[email\u00a0protected]<\/a>]<\/p>\n","protected":false},"excerpt":{"rendered":"Lee Sang-yeon, a professor of pediatric otorhinolaryngology at Seoul National University Hospital (SNUH), speaks to a patient at…\n","protected":false},"author":3,"featured_media":260500,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[26],"tags":[815,159,67,132,68],"class_list":{"0":"post-260499","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-united-states","11":"tag-unitedstates","12":"tag-us"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@us\/115280196688679326","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/posts\/260499","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/comments?post=260499"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/posts\/260499\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/media\/260500"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/media?parent=260499"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/categories?post=260499"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/us\/wp-json\/wp\/v2\/tags?post=260499"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}