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RPGR gene therapy presents challenges in cloning the coding sequence

INTRODUCTION: Currently, there are three Phase I/II clinical trials based on gene therapy ongoing to test different AAV.RPGR or deleted RPGR vectors on patients affected by X-linked retinitis pigmentosa. These three vectors differ in the adeno-associated viral (AAV) vector capsid used, and the codin...

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Detalhes bibliográficos
Publicado no:Expert Opin Biol Ther
Main Authors: De La Camara, Cristina Martinez-Fernandez, Cehajic-Kapetanovic, Jasmina, MacLaren, Robert E.
Formato: Artigo
Idioma:Inglês
Publicado em: 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7104355/
https://ncbi.nlm.nih.gov/pubmed/31612744
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/14712598.2020.1680635
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