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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
by
Wang, Raymond Y.
,
Movsesyan, Nina
,
Kan, Shih-hsin
,
Beydoun, Tammam
,
Taylor, Mery
,
Chang, Richard C.
,
Phillips, Dawn
,
Burke, Jenna
,
Gilmor, Michelle
,
Cho, Yoonjin
,
Falabella, Paulo
,
Pisani, Laura
Published in
Molecular genetics and metabolism
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First in-human intracisternal dosing of RGX-111 (adeno-associated virus 9/human α-L-iduronidase) for a 20-month-old child with mucopolysaccharidosis type I (MPS I): 1 year follow-u...
by
Wang, Raymond Y.
,
Beydoun, Tammam
,
Movsesyan, Nina
,
Kan, Shih-Hsin
,
Taylor, Mery
,
Stockton, Winnie
,
Nestrasil, Igor
,
Pukenas, Bryan
,
Falabella, Paulo
,
Cho, Yoonjin
,
Névoret, Marie-Laure
Published in
Molecular genetics and metabolism
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Molecular Genetics And Metabolism
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