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. Author manuscript; available in PMC: 2018 Jul 25.
Published in final edited form as: Expert Opin Orphan Drugs. 2018 Feb 27;6(3):167–177. doi: 10.1080/21678707.2018.1444476

Figure 3.

Figure 3

Schematic representation of the RPGR gene structure and the main alternatively spliced transcripts. Note that there is normally a splice donor site within the 5′ region of ORF15. In gene therapy, the cDNA encoding the gene is transcribed as a primary RNA transcript in the nucleus and may be subject to splicing. Hence disabling this splice donor site is also an essential part of codon optimisation.