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New AAV Vectors for Safe and Efficient Expression of Lysosomal Enzymes in the CNS

詳細技術說明
New AAV vectors that enable transgene expression at therapeutic levels for treatment of lysosomal and other disorders. These levels are achieved without the adverse events that arise due to secondary
*Abstract

New AAV vectors that enable transgene expression at therapeutic levels for treatment of lysosomal and other disorders. These levels are achieved without the adverse events that arise due to secondary effects of AAV-mediated product delivery. This newly engineered regulatory element can apply broadly to circumvent adverse effects of AAV therapy.

*IP Issue Date
None
*IP Type
Other Patent
國家
Not Available
申請號碼
None
國家/地區
美國

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