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Adeno-Associated Virus Serotype-Specific Inverted Terminal Repeat Sequence Role in Vector Transgene Expression

Hum Gene Ther. 2020; 
Earley LF, Conatser LM, Lue VM, Dobbins AL, Li C, , Hirsch ML, Samulski RJ, .
Products/Services Used Details Operation
PCR Cloning and Subcloning Methods Plasmid construction The ITR sequences from AAV serotypes 1‐4 & 6‐7 sequences were ordered from Genscript with unique restriction enzyme sites flanking the sequences for downstream cloning. Get A Quote

摘要

Adeno-associated viral vectors have been successfully used in laboratory and clinical settings for efficient gene delivery. In these vectors, 96% of the adeno-associated virus (AAV) genome is replaced with a gene cassette of interest, leaving only the 145 bp inverted terminal repeat (ITR) sequences. These cis-elements, primarily from AAV serotype 2, are required for genome rescue, replication, packaging, and vector persistence. Previous work from our lab and others have demonstrated that the AAV ITR2 sequence has inherent transcriptional activity, which may confound intended transgene expression in therapeutic applications. Currently, AAV capsids are extensively study for vector contribution; however, a compr... More

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