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Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
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机译:Aav9介导的基因疗法治疗I型黏多糖贮积病
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摘要
A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene and two or more immunosuppressants is provided herein. Also provided are methods useful for treating hIDUA deficiency (MPSI) and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.
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