Journal
HUMAN GENE THERAPY METHODS
Volume 30, Issue 6, Pages 206-213Publisher
MARY ANN LIEBERT, INC
DOI: 10.1089/hgtb.2019.276
Keywords
adeno-associated virus; inverted terminal repeats; ITR; quality control; AAV
Categories
Funding
- Australian National Health and Medical Research Council (NHMRC) [APP1108311, APP1161583]
- Department of Science and Higher Education of Ministry of National Defense, Republic of Poland [k/10/8047/DNiSW/T-WIHE/3]
- National Science Centre, Republic of Poland (OPUS 13) [UMO-2017/25/B/NZ1/02790]
- Rebecca Cooper Medical Research Foundation [PG2019449]
- Paediatrio Paediatric Precision Medicine Program [PPM1 K5116/RD274]
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Ongoing development of recombinant vectors based on adeno-associated virus (rAAV) is providing an increasingly powerful and widely used toolkit for gene transfer and genome editing applications. While conceptually simple, the system harbors considerable complexity that presents many potential pitfalls for the inexperienced user. The short inverted terminal repeats (ITRs) can prove to be particularly problematic during vector engineering due to inherent instability necessitating diligent quality control measures during vector manufacture. This is especially important from a clinical standpoint when consistent purity and potency are paramount, and all components of the system are rigorously scrutinized by regulatory agencies. Despite the discovery over 30 years ago that the AAV ITRs are the only cis-acting elements of the virus required for vector production, there is a scarcity of reviews specifically focused on these complex elements. This review provides an overview of the ITR with the dual purpose of acting as a user's guide in the application of AAV vector technology and as a roadmap for ongoing vector development and optimization.
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