دورية أكاديمية
RGX-121 gene therapy for the treatment of severe mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in-human study
العنوان: | RGX-121 gene therapy for the treatment of severe mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in-human study |
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المؤلفون: | Harmatz, Paul, Escolar, Maria, Ficicioglu, 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 S54 ; 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.2021.11.132 |
الإتاحة: | https://doi.org/10.1016/j.ymgme.2021.11.132Test https://api.elsevier.com/content/article/PII:S1096719221009501?httpAccept=text/xmlTest https://api.elsevier.com/content/article/PII:S1096719221009501?httpAccept=text/plainTest |
حقوق: | https://www.elsevier.com/tdm/userlicense/1.0Test/ |
رقم الانضمام: | edsbas.9D9306BF |
قاعدة البيانات: | BASE |
DOI: | 10.1016/j.ymgme.2021.11.132 |
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