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AAV-BASED GENE THERAPIES FOR TREATMENT OF AUTOIMMUNE DISEASES
AAV-BASED GENE THERAPIES FOR TREATMENT OF AUTOIMMUNE DISEASES
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机译:基于AAV的基因疗法治疗自身免疫性疾病
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摘要
Disclosed are AAV viral-based vector compositions useful in delivering a variety of nucleic acid segments, including those encoding therapeutic polypeptides to selected mammalian host cells for use in therapeutic autoimmune modalities, including, for example, the in vivo induction of immunological tolerance via a liver-directed AAV-based gene therapeutic regimen for treating and/or ameliorating autoimmune disorders such as multiple sclerosis. Further disclosed are nucleic acid segments encoding therapeutic polypeptides that have been codon-optimized for expression in human cells.
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