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First in-human, intracisternal dosing of RGX-111, an invest..:
Wang, Raymond Y.
;
Movsesyan, Nina
;
Kan, Shih-hsin
...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 108070 , 2024
Link:
https://doi.org/10.1016/j.ymgme.2023.108070
RT Journal T1
First in-human, intracisternal dosing of RGX-111, an investigational AAV gene therapy, for a 21-month-old child with mucopolysaccharidosis type I (MPS I): 3.5 year follow-up
UL https://suche.suub.uni-bremen.de/peid=cr-10.1016_j.ymgme.2023.108070&Exemplar=1&LAN=DE A1 Wang, Raymond Y. A1 Movsesyan, Nina A1 Kan, Shih-hsin A1 Beydoun, Tammam A1 Taylor, Mery A1 Chang, Richard C. A1 Phillips, Dawn A1 Burke, Jenna A1 Gilmor, Michelle A1 Cho, Yoonjin A1 Falabella, Paulo A1 Pisani, Laura PB Elsevier BV YR 2024 SN 1096-7192 JF Molecular Genetics and Metabolism VO 141 IS 2 SP 108070 LK http://dx.doi.org/https://doi.org/10.1016/j.ymgme.2023.108070 DO https://doi.org/10.1016/j.ymgme.2023.108070 SF ELIB - SuUB Bremen
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