Adeno-Associated VIRUS (AAV) Services

As cell and gene therapy research continues to advance across a wide-range of diseases, the need for effective vectors for successful therapy delivery has increased. Identified as an optimal vehicle, adeno-associated virus (AAV) plasmids are the leading viral vector used in in vivo gene therapy clinical trials due to their high efficiency and enhanced safety in humans.

As a global leader in genomics services, Azenta has developed unique AAV-ITR sequencing, AAV plasmid synthesis, and AAV plasmid preparation capabilities to support scientists in their in vivo gene therapy research. Azenta’s ground-breaking AAV services also include sequence verification and correction of inverted terminal repeat (ITR) regions, which are crucial for rAAV packaging production.

AAV Services

AAV-ITR Sequencing

Utilizes Sanger protocols to sequence-confirm difficult ITR regions, expediting screening and validation of lead candidates. Whole AAV plasmid sequencing through primer walking is also available.

AAV Plasmid Preparation

Ensures delivery of intact ITR regions while providing superior quality for mini-to-giga scale AAV vectors.

AAV Plasmid Synthesis

Enables synthesis and cloning of transgene expression cassettes into custom AAV vectors. Includes ITR correction to synthesize and clone correct ITR sequences.

AAV Genome Sequencing

Reimagine quality control standards for rAAV vectors by employing long-read (>10kb) NGS to examine genome integrity, detect heterogeneity, and reveal packaging attributes. Analyze host response utilizing RNA-seq and a variety of other proprietary deep sequencing approaches.

AAV ADVANTAGES

Low cell toxicity as it does not integrate into host genome

Low immunogenicity and non-pathogenic with minimal immune host response

High levels of in vivo gene expression

High transduction efficiency across a broad range of target tissues

AAV SYSTEM

Adeno-associated virus (AAV) is one of the most actively investigated gene therapy vehicles. AAV has a linear single-stranded DNA (ssDNA) genome of approximately 4.7 kilobases (kb), with two 145 nucleotide-long inverted terminal repeats (ITR) at the termini which are crucial for rAAV production.

Figure 1. An AAV transfer plasmid containing the modified/transgene expression cassette with intact ITRs is generated and cloned into a plasmid along with Rep/Cap and helper plasmids, which are co-transfected into cell lines for virus packaging and rAAV production.

Features & Benefits

Rapid Turnaround Times

Starting at just 5 business days
Real-Time Project Updates

Through our online system
Ph.D.-Level Project Managers

Available at each step

End-to-end DNA AAV solutions with ITR sequencing verification, transgene synthesis, and plasmid preparation

Low immunogenicity and non-pathogenic with minimal immune host response

Robust quality control ensures AAV plasmids are delivered with intact ITRs confirmed via AAV-ITR sequencing

Convenient online ordering directly through your CLIMS account for all services

Technical Resources

Webinar: Effective and High-Quality Solutions for AAV-Based Therapies

In this webinar, Azenta’s Dr. Eric Zhao will present our unique AAV-ITR Sanger sequencing and AAV plasmid preparation capabilities that maintain ITR integrity for downstream virus production, as well as our next generation sequencing-enabled QC of packaged rAAV vectors and host genomes using long-read and short-read sequencing technologies.

Tech Note: Reading Through the Inverted Terminal Repeats (ITRs) of Adeno-associated Virus (AAV)

Azenta has developed a high-quality, direct Sanger sequencing method that reads through both intact and commonly mutated inverted terminal repeat (ITR) regions of adeno-associated virus (AAV). This method is an effective tool for assessing the integrity of ITRs in AAV plasmids.