Large gene delivery to the retina with AAV vectors: are we there yet?

Ivana Trapani^{1

2}, Patrizia Tornabene^{1

2}, Alberto Auricchio^{3

4}

Affiliations

¹ Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy.
² Medical Genetics, Department of Translational Medicine, Federico II University, Naples, Italy.
³ Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy. auricchio@tigem.it.
⁴ Department of Advanced Biomedicine, Federico II University, Naples, Italy. auricchio@tigem.it.

Ivana Trapani et al. Gene Ther. 2021 May.

. 2021 May;28(5):220-222.

doi: 10.1038/s41434-020-0174-4. Epub 2020 Jul 13.

Ivana Trapani^{1

2}, Patrizia Tornabene^{1

2}, Alberto Auricchio^{3

4}

¹ Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy.
² Medical Genetics, Department of Translational Medicine, Federico II University, Naples, Italy.
³ Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy. auricchio@tigem.it.
⁴ Department of Advanced Biomedicine, Federico II University, Naples, Italy. auricchio@tigem.it.

No abstract available

References

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1. Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18:358–78. - DOI
1. Trapani I, Auricchio A. Seeing the light after 25 years of retinal gene therapy. Trends Mol Med. 2018;24:669–81. - DOI
1. Chamberlain K, Riyad JM, Weber T. Expressing transgenes that exceed the packaging capacity of adeno-associated virus capsids. Hum Gene Ther Methods. 2016;27:1–12. - DOI