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, AAV packaging 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.

Enhancing Gene Transfer Efficiency:
Viral vs. Non-Viral Methods

Friday, September 20, 2024 | 11:00AM EDT

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 Packaging

Unlock the full potential of cell or gene therapy by overcoming the instability of AAV vectors with GENEWIZ's AAV packaging workflow. Our proprietary AAV plasmid synthesis process offers industry-leading project quality, completion time, and flexibility, ensuring researchers can harness the powerful benefits of this technology effectively.

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

Proprietary technologies with optimized workflows specifically designed for AAV vectors

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

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.

Tech Note: An Efficient and Reliable Approach to AAV Packaging

While AAV is a powerful vehicle for gene delivery, unstable ITRs threaten the fidelity of rAAV genomes. Loss of ITR integrity diminishes the packaging efficiency of rAAV particles and thus viral titer. In this tech note, discover the effect of partial and full ITR deletions on AAV packaging and how to reliably identify and effectively correct them.