--- title: Tackling rare genetic disorders with patient-focused science description: A sixth-year PhD candidate at MIT's McGovern Institute for Brain Research is developing a CRISPR-based gene therapy targeting SYNGAP1 haploinsufficiency, a... canonical: https://daily.dev/posts/tackling-rare-genetic-disorders-with-patient-focused-science-krlenyafo twitter:card: summary_large_image twitter:site: @dailydotdev og:type: website og:site_name: daily.dev og:title: Tackling rare genetic disorders with patient-focused science | daily.dev og:description: A sixth-year PhD candidate at MIT's McGovern Institute for Brain Research is developing a CRISPR-based gene therapy targeting SYNGAP1 haploinsufficiency, a... og:url: https://daily.dev/posts/tackling-rare-genetic-disorders-with-patient-focused-science-krlenyafo og:image: https://api.daily.dev/og/posts/KrlEnYAFO.png og:image:alt: Tackling rare genetic disorders with patient-focused science 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. # Tackling rare genetic disorders with patient-focused science **[MIT News](https://daily.dev/sources/mit)** · 5 min read · 0 upvotes · 0 comments ## Summary A sixth-year PhD candidate at MIT's McGovern Institute for Brain Research is developing a CRISPR-based gene therapy targeting SYNGAP1 haploinsufficiency, a rare genetic disorder causing childhood-onset epilepsy and intellectual disabilities. Building on related gene therapy work for Phelan-McDermid syndrome that is now in clinical trials, mouse testing of the SYNGAP1 therapy has alleviated seizures and behavioral symptoms. The work is supported by MIT's Rare Brain Disorders Nexus initiative, and the researcher also teaches molecular neurobiology lab courses, having received the Goodwin Medal for teaching excellence. ## Full article daily.dev links to this article rather than hosting it. Read it at the original source: ## Similar posts on daily.dev - [New MIT initiative seeks to transform rare brain disorders research](https://daily.dev/posts/new-mit-initiative-seeks-to-transform-rare-brain-disorders-research-tlk98feby) · 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 - [Targeting Allele-Specific Faulty MRNA In SCNA2 Mutation Patients](https://daily.dev/posts/targeting-allele-specific-faulty-mrna-in-scna2-mutation-patients-uen0mak1v) · Hackaday · 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 - [How AI is helping solve the labor issue in treating rare diseases](https://daily.dev/posts/how-ai-is-helping-solve-the-labor-issue-in-treating-rare-diseases-rcbudxyor) · TechCrunch · 1 upvotes · 0 comments --- Tags: [#biotech](https://daily.dev/tags/biotech) [View this post on daily.dev](https://daily.dev/posts/tackling-rare-genetic-disorders-with-patient-focused-science-krlenyafo) ```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":"Tackling rare genetic disorders with patient-focused science","url":"https://daily.dev/posts/tackling-rare-genetic-disorders-with-patient-focused-science-krlenyafo","mainEntityOfPage":{"@type":"WebPage","@id":"https://daily.dev/posts/tackling-rare-genetic-disorders-with-patient-focused-science-krlenyafo"},"datePublished":"2026-08-18T04:04:12.269Z","dateModified":"2026-08-18T04:04:32.644Z","description":"A sixth-year PhD candidate at MIT's McGovern Institute for Brain Research is developing a CRISPR-based gene therapy targeting SYNGAP1 haploinsufficiency, a...","image":"https://media.daily.dev/image/upload/f_auto,q_auto/v1/posts/a579da08cd8da0a8722279fd91b73265?_a=AQAEuop","thumbnailUrl":"https://media.daily.dev/image/upload/f_auto,q_auto/v1/posts/a579da08cd8da0a8722279fd91b73265?_a=AQAEuop","isAccessibleForFree":true,"articleSection":"MIT News","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":"MIT News","logo":"https://media.daily.dev/image/upload/t_logo,f_auto/v1/logos/881e20a40aa44b12ba4bd27f5c14289d","url":"https://daily.dev/sources/mit"},"commentCount":0,"discussionUrl":"https://daily.dev/posts/tackling-rare-genetic-disorders-with-patient-focused-science-krlenyafo","interactionStatistic":[{"@type":"InteractionCounter","interactionType":{"@type":"LikeAction"},"userInteractionCount":0},{"@type":"InteractionCounter","interactionType":{"@type":"CommentAction"},"userInteractionCount":0}],"keywords":"biotech","timeRequired":"PT5M"} {"@context":"https://schema.org","@type":"BreadcrumbList","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https://daily.dev"},{"@type":"ListItem","position":2,"name":"MIT News","item":"https://daily.dev/sources/mit"},{"@type":"ListItem","position":3,"name":"Tackling rare genetic disorders with patient-focused science"}]} ```