دورية أكاديمية
RGX-111 gene therapy for the treatment of severe mucopolysaccharidosis type I (MPS I): Interim analysis of data from the first in-human study
| العنوان: | RGX-111 gene therapy for the treatment of severe mucopolysaccharidosis type I (MPS I): Interim analysis of data from the first in-human study |
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| المؤلفون: | Wang, Raymond, Ficiciolu, Can, Giugliani, Roberto, Nevoret, Marie-Laure, Fiscella, Michele, Yang, Lin, Cho, Yoonjin, Phillips, Dawn, Falabella, Paulo |
| المصدر: | Molecular Genetics and Metabolism ; volume 135, issue 2, page S126 ; ISSN 1096-7192 |
| بيانات النشر: | Elsevier BV |
| سنة النشر: | 2022 |
| المجموعة: | ScienceDirect (Elsevier - Open Access Articles via Crossref) |
| مصطلحات موضوعية: | Endocrinology, Genetics, Molecular Biology, Biochemistry, Endocrinology, Diabetes and Metabolism |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| DOI: | 10.1016/j.ymgme.2021.11.335 |
| الإتاحة: | https://doi.org/10.1016/j.ymgme.2021.11.335Test https://api.elsevier.com/content/article/PII:S1096719221011537?httpAccept=text/xmlTest https://api.elsevier.com/content/article/PII:S1096719221011537?httpAccept=text/plainTest |
| حقوق: | https://www.elsevier.com/tdm/userlicense/1.0Test/ |
| رقم الانضمام: | edsbas.734772 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1016/j.ymgme.2021.11.335 |
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