Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy.

@article{Yan2000TranssplicingVE,
  title={Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy.},
  author={Ziying Yan and Yaping Zhang and Dongsheng Duan and John F. Engelhardt},
  journal={Proceedings of the National Academy of Sciences of the United States of America},
  year={2000},
  volume={97 12},
  pages={6716-21}
}
Adeno-associated viral (AAV) vectors have demonstrated considerable promise for gene therapy of inherited diseases. However, with a packaging size of <5 kb, applications have been limited to relatively small disease genes. Based on the finding that AAV genomes undergo intermolecular circular concatamerization after transduction in muscle, we have developed a paradigm to increase the size of delivered transgenes with this vector through trans-splicing between two independent vectors… CONTINUE READING

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