Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa

Cristina Martinez-Fernandez de la Camaraa Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, John Radcliffe Hospital, Level 5 & 6, West Wing, Headley Way, OX3 9DU, Oxford, UK;b Oxford Eye Hospital, Oxford University Hospitals NHS Trust, John Radcliffe Hospital, West Wing, Headley Way, OX3 9DU, Oxford, UKView further author information

Jasmina Cehajic-Kapetanovica Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, John Radcliffe Hospital, Level 5 & 6, West Wing, Headley Way, OX3 9DU, Oxford, UK;b Oxford Eye Hospital, Oxford University Hospitals NHS Trust, John Radcliffe Hospital, West Wing, Headley Way, OX3 9DU, Oxford, UKView further author information

Robert E. MacLarena Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, John Radcliffe Hospital, Level 5 & 6, West Wing, Headley Way, OX3 9DU, Oxford, UK;b Oxford Eye Hospital, Oxford University Hospitals NHS Trust, John Radcliffe Hospital, West Wing, Headley Way, OX3 9DU, Oxford, UKCorrespondence[email protected]
View further author information

Figures & data

Table 1. Cell therapy clinical trials.

Download CSV Display Table

Table 2. Gene therapy clinical trials.

Download CSV Display Table

Figure 1. RPGR^ORF15 is located in the connecting cilia of rods and cones. Its proposed function is to enable the transport of different molecules along the connecting cilia, for which its glutamylation and its interaction with other proteins seem to be essential.

Figure 2. Recombinant AAV.RPGR vectors currently being tested in human clinical trials. The three AAV vectors administered in the subretinal space use the human GRK1 promoter to drive the expression of RPGR^ORF15. The vectors developed by Biogen Inc., AGTC and 4D Molecular Therapeutics contain a codon-optimized (co) version of the human RPGRORF15 gene whilst the vector developed by MeiraGTx contains a shortened version of the human RPGR^ORF15. The AAV serotype capsids used to develop these vectors are AAV8 (Biogen), AAV5 (MeiraGTx), an AAV2 variant, with three tyrosine to phenylalanine mutations (AAV2tYF) (AGTC), and an AAV capsid variant named 4D-R100 suitable for intravitreal administration (4D Molecular Therapeutics).

Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa

Table 1. Cell therapy clinical trials.

Table 2. Gene therapy clinical trials.

Information for

Open access

Opportunities

Help and information

Your download is now in progress and you may close this window

Login or register to access this feature

Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa

Figures & data

Table 1. Cell therapy clinical trials.

Table 2. Gene therapy clinical trials.

Related research

To cite this article:

Download citation

Information for

Open access

Opportunities

Help and information

Keep up to date