Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina-Reference-Cited by-同舟云学术

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

Published:2019-05-15 Issue:492 Volume:11 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Tornabene Patrizia¹^ORCID,Trapani Ivana¹²^ORCID,Minopoli Renato¹,Centrulo Miriam¹,Lupo Mariangela¹,de Simone Sonia¹,Tiberi Paola¹^ORCID,Dell’Aquila Fabio¹,Marrocco Elena¹,Iodice Carolina¹,Iuliano Antonella¹,Gesualdo Carlo³,Rossi Settimio³,Giaquinto Laura¹^ORCID,Albert Silvia⁴,Hoyng Carel B.⁵,Polishchuk Elena¹^ORCID,Cremers Frans P. M.⁵^ORCID,Surace Enrico M.¹²,Simonelli Francesca³,De Matteis Maria A.¹⁶^ORCID,Polishchuk Roman¹^ORCID,Auricchio Alberto¹⁷^ORCID

Affiliation:

1. Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.

2. Medical Genetics, Department of Translational Medicine, Federico II University, 80131 Naples, Italy.

3. Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, University of Campania L. Vanvitelli, 80131 Naples, Italy.

4. Department of Human Genetics and Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 Nijmegen, Netherlands.

5. Department of Ophthalmology and Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 Nijmegen, Netherlands.

6. Department of Molecular Medicine and Medical Biotechnology, Federico II University, 80131 Naples, Italy.

7. Department of Advanced Biomedicine, Federico II University, 80131 Naples, Italy.

Abstract

Adeno-associated viral vectors reconstitute large proteins in the retina via intein-mediated protein trans-splicing.

Funder

Universita degli Studi di Napoli Federico II

European Research Council

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference35 articles.

1. FDA approves hereditary blindness gene therapy

2. Seeing the Light after 25 Years of Retinal Gene Therapy

3. The Future Looks Brighter After 25 Years of Retinal Gene Therapy

4. Effective delivery of large genes to the retina by dual AAV vectors

5. Expanding AAV Packaging Capacity with Trans-splicing or Overlapping Vectors: A Quantitative Comparison

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