Human Gene Therapy
 2024
DOI: 10.1089/hum.2024.096
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Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice

Angélica María Herreño-Pachón,
Kazuki Sawamoto,
Molly Stapleton
et al.
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