Provided herein are gene therapy methods for the treatment of mucopolysaccharidosis type IV A (MPS IV A) involving the use of recombinant adeno-associated viruses (rAAVs) to deliver human N-acetylgalactosamine-6-sulfate sulfatase (hGALNS) to the bone of a human subject diagnosed with MPS IVA. Also provided herein are rAAVs that can be used in the gene therapy methods and methods of making such rAAVs.
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机译:本文提供了用于治疗粘性多种素的基因治疗方法IV A(MPS IV A),涉及使用重组腺相关病毒(RAAVS)将人N-乙酰甘酰胺-6-硫酸盐硫酸酯(HGALNS)递送至a的骨人类受试者被诊断患有MPS IVA。本文还提供了可用于基因治疗方法和制造此类RAAV的方法的rAAV。
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