{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,8]],"date-time":"2026-06-08T07:58:25Z","timestamp":1780905505249,"version":"3.54.1"},"reference-count":77,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,6,8]],"date-time":"2026-06-08T00:00:00Z","timestamp":1780876800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,6,8]],"date-time":"2026-06-08T00:00:00Z","timestamp":1780876800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/501100012166","name":"National Basic Research Program of China","doi-asserted-by":"publisher","award":["2024YFC3408100"],"award-info":[{"award-number":["2024YFC3408100"]}],"id":[{"id":"10.13039\/501100012166","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Neuroprotection"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)\u2010based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR\u2010associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)\u2010encoded template. It enables precise base substitutions, small insertions, and deletions without introducing double\u2010strand breaks, thereby expanding the range of correctable mutations while reducing undesired repair outcomes. This technology offers a promising strategy for genomic correction in the nervous system. Here, we review the development of prime editing, its mechanistic rationale, and emerging preclinical evidence that supports its application in neuropsychiatric disorders. We discuss key biological and technological barriers, including limited editing efficiency in post\u2010mitotic neurons, complex pegRNA design, reverse transcription\u2010related errors, vector payload limitations, and blood\u2010brain barrier (BBB) penetration. Nevertheless, in vitro and in vivo studies have demonstrated proof\u2010of\u2010concept correction and functional rescue in several monogenic neurodevelopmental disorders. Advances such as split\u2010adeno\u2010associated virus (AAV) systems, lipid nanoparticles, engineered peptides, and compact Cas variants are actively expanding their therapeutic potential. Further clinical translation will rely on improved editors with guide engineering, BBB\u2010penetrant and neuron\u2010targeted delivery platforms, transient or cell\u2010type\u2010specific expression strategies, and comprehensive genome\u2010wide safety evaluations.<\/jats:p>","DOI":"10.1002\/nep3.70041","type":"journal-article","created":{"date-parts":[[2026,6,8]],"date-time":"2026-06-08T07:15:11Z","timestamp":1780902911000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Prime editing in neuropsychiatric disorders: From mutation\u2010specific target selection to clinical translation"],"prefix":"10.1002","author":[{"given":"Tianshan","family":"Ji","sequence":"first","affiliation":[{"name":"Department of Physiology, School of Basic Medical Science Nanjing Medical University Nanjing Jiangsu China"},{"name":"Jiangsu Provincial Key Laboratory of Biological Therapy for Organ Failure, School of Basic Medical Science Nanjing Medical University Nanjing Jiangsu China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuan","family":"Zhang","sequence":"additional","affiliation":[{"name":"Tianyuan Honors School Nanjing Medical University Nanjing Jiangsu China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jinyi","family":"Zhao","sequence":"additional","affiliation":[{"name":"The First School of Clinical Medicine Nanjing Medical University Nanjing Jiangsu China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yiping","family":"Lu","sequence":"additional","affiliation":[{"name":"The Second School of Clinical Medicine Nanjing Medical University Nanjing Jiangsu China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2505-7295","authenticated-orcid":false,"given":"Chengkun","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Physiology, School of Basic Medical Science Nanjing Medical University Nanjing Jiangsu China"},{"name":"Jiangsu Provincial Key Laboratory of Biological Therapy for Organ Failure, School of Basic Medical Science Nanjing Medical University Nanjing Jiangsu China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,6,8]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"crossref","DOI":"10.1016\/j.preteyeres.2022.101132","article-title":"In vivo application of base and prime editing to treat inherited retinal diseases","volume":"94","author":"Jo DH","year":"2023","journal-title":"Prog Retinal Eye Res"},{"issue":"7","key":"e_1_2_12_3_1","doi-asserted-by":"crossref","first-page":"692","DOI":"10.1016\/j.tibtech.2020.10.010","article-title":"Next\u2010Generation CRISPR technologies and their applications in gene and cell therapy","volume":"39","author":"Zeballos C. 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