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1.
A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia.
Sci Adv
; 7(44): eabj5018, 2021 Oct 29.
Artículo
en Inglés
| MEDLINE | ID: mdl-34705504
2.
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
Nat Commun
; 12(1): 6393, 2021 11 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-34737297
3.
Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.
EBioMedicine
; 61: 103052, 2020 Nov.
Artículo
en Inglés
| MEDLINE | ID: mdl-33039711
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