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Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage.
Hum Gene Ther. 2024 Apr;35(7-8):256-268. doi: 10.1089/hum.2023.177. Epub 2024 Feb 2.
Hum Gene Ther. 2024.
PMID: 38085235
Free PMC article.
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