Pub Date : 2025-02-01DOI: 10.1016/j.ymgme.2024.108631
Pratikshya Adhikari, Neil Sud, Tierra Bobo, Haiyan Fu
{"title":"AAV9-based gene replacement therapy targeting the root cause for the treatment of MPS IIID in mice","authors":"Pratikshya Adhikari, Neil Sud, Tierra Bobo, Haiyan Fu","doi":"10.1016/j.ymgme.2024.108631","DOIUrl":"10.1016/j.ymgme.2024.108631","url":null,"abstract":"","PeriodicalId":18937,"journal":{"name":"Molecular genetics and metabolism","volume":"144 2","pages":"Article 108631"},"PeriodicalIF":3.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143172951","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-01DOI: 10.1016/j.ymgme.2024.108680
Chong Kun Cheon, Jin Hur, Soo Yong Lee
{"title":"Evaluation of potential drug development and therapeutic approaches for mucopolysaccharidosis type II","authors":"Chong Kun Cheon, Jin Hur, Soo Yong Lee","doi":"10.1016/j.ymgme.2024.108680","DOIUrl":"10.1016/j.ymgme.2024.108680","url":null,"abstract":"","PeriodicalId":18937,"journal":{"name":"Molecular genetics and metabolism","volume":"144 2","pages":"Article 108680"},"PeriodicalIF":3.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143173263","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-01DOI: 10.1016/j.ymgme.2024.108688
John W. Collyer , Deepa Rajan , Michele Poe , Paul Szabolcs , Maria L. Escolar
{"title":"Neurodevelopmental benefits of early hematopoietic stem cell transplantation in mucopolysaccharidosis type II","authors":"John W. Collyer , Deepa Rajan , Michele Poe , Paul Szabolcs , Maria L. Escolar","doi":"10.1016/j.ymgme.2024.108688","DOIUrl":"10.1016/j.ymgme.2024.108688","url":null,"abstract":"","PeriodicalId":18937,"journal":{"name":"Molecular genetics and metabolism","volume":"144 2","pages":"Article 108688"},"PeriodicalIF":3.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143173268","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-01DOI: 10.1016/j.ymgme.2024.108716
Jacob Favret, Meghana Kushawa, Daesung Shin
{"title":"Dissecting the role of oligodendroglial NFkB signaling in the pathogenesis of Krabbe disease","authors":"Jacob Favret, Meghana Kushawa, Daesung Shin","doi":"10.1016/j.ymgme.2024.108716","DOIUrl":"10.1016/j.ymgme.2024.108716","url":null,"abstract":"","PeriodicalId":18937,"journal":{"name":"Molecular genetics and metabolism","volume":"144 2","pages":"Article 108716"},"PeriodicalIF":3.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143173720","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-01DOI: 10.1016/j.ymgme.2024.108710
Hatim Y. Ebrahim , Uma Ramaswami , Derralynn A. Hughes
{"title":"Audit of patients screened for lysosomal disorders at the Royal Free London Hospital","authors":"Hatim Y. Ebrahim , Uma Ramaswami , Derralynn A. Hughes","doi":"10.1016/j.ymgme.2024.108710","DOIUrl":"10.1016/j.ymgme.2024.108710","url":null,"abstract":"","PeriodicalId":18937,"journal":{"name":"Molecular genetics and metabolism","volume":"144 2","pages":"Article 108710"},"PeriodicalIF":3.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143174259","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-01DOI: 10.1016/j.ymgme.2024.108707
Mireia Del Toro Riera, Maria Luz Uria Oficialdegui, Lucy D. Dougherty De Miguel, Andrea Olivares, Melissa Panesso, Ana Felipe Rucian, Maria Camprodon-Gomez, Cristina Diaz-de-Heredia
{"title":"Long-term outcome of mucopolysaccharidosis type I (Hurler syndrome) patients after hematopoietic cell transplantation","authors":"Mireia Del Toro Riera, Maria Luz Uria Oficialdegui, Lucy D. Dougherty De Miguel, Andrea Olivares, Melissa Panesso, Ana Felipe Rucian, Maria Camprodon-Gomez, Cristina Diaz-de-Heredia","doi":"10.1016/j.ymgme.2024.108707","DOIUrl":"10.1016/j.ymgme.2024.108707","url":null,"abstract":"","PeriodicalId":18937,"journal":{"name":"Molecular genetics and metabolism","volume":"144 2","pages":"Article 108707"},"PeriodicalIF":3.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143132156","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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