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Commentary

Recent tissue engineering-based advances for effective rAAV-mediated gene transfer in the musculoskeletal system

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Pages 175-188 | Received 26 Apr 2016, Accepted 02 May 2016, Published online: 24 May 2016

Figures & data

Figure 1. Concepts for rAAV-mediated gene transfer using tissue engineering approaches in the musculoskeletal system. (A) Genomic organization of rAAV vectors. Classical rAAV vector with 2 inverted terminal repeats (ITRs) at either end of a transgene cassette (heterelogous promoter, gene of interest, intron/polyA signal). The arrows show the viral transcription promoter. (B) Principal tissue engineering strategies for rAAV mediated-gene transfer in the musculoskeletal system. rAAV can be encapsulated in different biomaterials such as hydrogels or polymeric micelles to achieve a controlled release profile at the site of injury. The vectors may be delivered ex vivo by genetically modification of cells that are subsequently seeded onto a matrix and implanted in the recipient. Different patient-related materials including bone marrow aspirates (BMA) and allografts can be endowed with biological factors enhancing cell/tissue reparative processes via rAAV-mediated gene transfer. Polymers can be used to overcome rAAV physiological barriers when administered through classical routes to achieve an efficient gene transfer in the target location.

Figure 1. Concepts for rAAV-mediated gene transfer using tissue engineering approaches in the musculoskeletal system. (A) Genomic organization of rAAV vectors. Classical rAAV vector with 2 inverted terminal repeats (ITRs) at either end of a transgene cassette (heterelogous promoter, gene of interest, intron/polyA signal). The arrows show the viral transcription promoter. (B) Principal tissue engineering strategies for rAAV mediated-gene transfer in the musculoskeletal system. rAAV can be encapsulated in different biomaterials such as hydrogels or polymeric micelles to achieve a controlled release profile at the site of injury. The vectors may be delivered ex vivo by genetically modification of cells that are subsequently seeded onto a matrix and implanted in the recipient. Different patient-related materials including bone marrow aspirates (BMA) and allografts can be endowed with biological factors enhancing cell/tissue reparative processes via rAAV-mediated gene transfer. Polymers can be used to overcome rAAV physiological barriers when administered through classical routes to achieve an efficient gene transfer in the target location.

Table 1. Tissue engineering approaches for rAAV gene transfer in articular cartilage.

Table 2. Tissue engineering approaches for rAAV gene transfer in bone.

Table 3. Tissue engineering approaches for rAAV gene transfer in tendons and muscles.

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