RPGR gene therapy presents challenges in cloning the coding sequence

<p><strong>Introduction:</strong> 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 (...

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Bibliografske podrobnosti
Main Authors: Martinez-Fernandez De La Camara, C, Cehajic-Kapetanovic, J, MacLaren, RE
Format: Journal article
Jezik:English
Izdano: Taylor and Francis 2019