Custom rAAV Vectors

Vectors Built to Your Specifications

REGENX will produce rAAV preps with your choice of expression cassette (e.g., promoter, transgene) and serotype. One plasmid with your expression cassette can be used to produce multiple different rAAV serotypes. A single rAAV prep will contain approximately 5×1012 GC (genome copies) of rAAV. If you need larger amounts, multiple preps can be generated in parallel, pooled, and quality controlled.

Elements of the expression cassette:

  • The promoter may be one that works in all tissues, a tissue-specific promoter, or a regulatable promoter. In the case of shRNA expression, a pol II promoter may be optimal.
  • The transgene can be a cDNA of a wild-type sequence or a disease-causing mutant sequence, an antibody or antibody fragment, a reporter gene (see below) or antisense RNA or shRNA sequence. Note that secreted proteins may be expressed in tissues other than where normally expressed – for example, liver can be used to express large amounts of circulating secreted proteins. For intracellular or cell surface proteins, and for shRNAs, it may be critical to express those in the tissues and cell types that normally express those proteins.
  • Polyadenylation (polyA) signal. This is required for processing of messenger RNA.

REGENX can provide you with plasmids containing your desired promoter and polyA sequence, for you to insert your cDNA, or REGENX can clone the expression cassette for you.