--- title: Targeting Allele-Specific Faulty MRNA In SCNA2 Mutation... description: A recent Nature study demonstrated allele-specific antisense oligonucleotide (ASO) therapy in two boys with SCN2A mutations causing severe epilepsy and... canonical: https://daily.dev/posts/targeting-allele-specific-faulty-mrna-in-scna2-mutation-patients-uen0mak1v twitter:card: summary_large_image twitter:site: @dailydotdev og:type: website og:site_name: daily.dev og:title: Targeting Allele-Specific Faulty MRNA In SCNA2 Mutation Patients | daily.dev og:description: A recent Nature study demonstrated allele-specific antisense oligonucleotide (ASO) therapy in two boys with SCN2A mutations causing severe epilepsy and... og:url: https://daily.dev/posts/targeting-allele-specific-faulty-mrna-in-scna2-mutation-patients-uen0mak1v og:image: https://api.daily.dev/og/posts/uEn0MaK1v.png og:image:alt: Targeting Allele-Specific Faulty MRNA In SCNA2 Mutation Patients og:image:width: 1200 og:image:height: 630 og:locale: en --- > ## Documentation Index > Fetch the complete documentation index at: https://daily.dev/llms.txt > Use this file to discover all available pages before exploring further. # Targeting Allele-Specific Faulty MRNA In SCNA2 Mutation Patients **[Hackaday](https://daily.dev/sources/hackaday)** · 3 min read · 0 upvotes · 0 comments ## Summary A recent Nature study demonstrated allele-specific antisense oligonucleotide (ASO) therapy in two boys with SCN2A mutations causing severe epilepsy and developmental disorders. By targeting only the mutated allele's faulty mRNA — leaving the healthy copy intact — one patient achieved complete seizure elimination and the other saw significant reduction. Both patients also showed improvements in language, motor skills, and behavior. The approach offers a middle ground between symptom management and direct DNA editing, with potential for early intervention in affected infants. CRISPR-based gene correction in mouse models is also being explored as a more permanent solution. ## Full article daily.dev links to this article rather than hosting it. Read it at the original source: ## Similar posts on daily.dev - [Tackling rare genetic disorders with patient-focused science](https://daily.dev/posts/tackling-rare-genetic-disorders-with-patient-focused-science-krlenyafo) · MIT News · 0 upvotes · 0 comments - [A new CRISPR startup is betting regulators will ease up on gene-editing](https://daily.dev/posts/a-new-crispr-startup-is-betting-regulators-will-ease-up-on-gene-editing-xm4dkkwb1) · MIT Technology Review · 0 upvotes · 0 comments - [Rett syndrome study highlights potential for personalized treatments](https://daily.dev/posts/rett-syndrome-study-highlights-potential-for-personalized-treatments-khso7nvfz) · MIT News · 0 upvotes · 0 comments - [New insights into a hidden process that protects cells from harmful mutations](https://daily.dev/posts/new-insights-into-a-hidden-process-that-protects-cells-from-harmful-mutations-ba3jd8gza) · MIT News · 0 upvotes · 0 comments - [A better DNA material for genetic medicine](https://daily.dev/posts/a-better-dna-material-for-genetic-medicine-sobm9okhn) · MIT News · 0 upvotes · 0 comments --- [View this post on daily.dev](https://daily.dev/posts/targeting-allele-specific-faulty-mrna-in-scna2-mutation-patients-uen0mak1v) ```json {"@context":"https://schema.org","@graph":[{"@type":"Organization","@id":"https://daily.dev/#organization","name":"daily.dev","url":"https://daily.dev","logo":{"@type":"ImageObject","url":"https://daily.dev/apple-touch-icon.png","width":180,"height":180},"sameAs":["https://twitter.com/dailydotdev","https://github.com/dailydotdev","https://www.linkedin.com/company/daily-dev-ltd"]},{"@type":"WebSite","@id":"https://daily.dev/#website","url":"https://daily.dev","name":"daily.dev","publisher":{"@id":"https://daily.dev/#organization"},"potentialAction":{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https://daily.dev/search?q={search_term_string}"},"query-input":"required name=search_term_string"}}]} {"@context":"https://schema.org","@type":"TechArticle","headline":"Targeting Allele-Specific Faulty MRNA In SCNA2 Mutation Patients","url":"https://daily.dev/posts/targeting-allele-specific-faulty-mrna-in-scna2-mutation-patients-uen0mak1v","mainEntityOfPage":{"@type":"WebPage","@id":"https://daily.dev/posts/targeting-allele-specific-faulty-mrna-in-scna2-mutation-patients-uen0mak1v"},"datePublished":"2026-07-24T02:10:38.939Z","dateModified":"2026-07-24T02:12:22.086Z","description":"A recent Nature study demonstrated allele-specific antisense oligonucleotide (ASO) therapy in two boys with SCN2A mutations causing severe epilepsy and...","image":"https://media.daily.dev/image/upload/f_auto,q_auto/v1/posts/10a6ee2cf94614478be332cf3f1d7bd1?_a=AQAEuop","thumbnailUrl":"https://media.daily.dev/image/upload/f_auto,q_auto/v1/posts/10a6ee2cf94614478be332cf3f1d7bd1?_a=AQAEuop","isAccessibleForFree":true,"articleSection":"Hackaday","inLanguage":"en","publisher":{"@type":"Organization","name":"daily.dev","url":"https://daily.dev","logo":{"@type":"ImageObject","url":"https://daily.dev/apple-touch-icon.png","width":180,"height":180}},"author":{"@type":"Organization","name":"Hackaday","logo":"https://media.daily.dev/image/upload/s--JhDfhy70--/f_auto,q_auto/v1767541814/logos/hackaday","url":"https://daily.dev/sources/hackaday"},"commentCount":0,"discussionUrl":"https://daily.dev/posts/targeting-allele-specific-faulty-mrna-in-scna2-mutation-patients-uen0mak1v","interactionStatistic":[{"@type":"InteractionCounter","interactionType":{"@type":"LikeAction"},"userInteractionCount":0},{"@type":"InteractionCounter","interactionType":{"@type":"CommentAction"},"userInteractionCount":0}],"keywords":"","timeRequired":"PT3M"} {"@context":"https://schema.org","@type":"BreadcrumbList","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https://daily.dev"},{"@type":"ListItem","position":2,"name":"Hackaday","item":"https://daily.dev/sources/hackaday"},{"@type":"ListItem","position":3,"name":"Targeting Allele-Specific Faulty MRNA In SCNA2 Mutation Patients"}]} ```