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Gene therapy for choroideremia using an adeno-associated viral vector encoding Rab escort protein 1: the REGENERATE open-label trial

Journal article

Cehajic-Kapetanovic J. et al, (2024), Efficacy and Mechanism Evaluation, 1 - 59

IMPG2-Related Maculopathy.

Journal article

Birtel J. et al, (2024), Am J Ophthalmol, 258, 32 - 42

Optical Coherence Tomography Feature of Retinoschisis in CRB1-Associated Maculopathy

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Bellingrath J-S. et al, (2024), JAMA Ophthalmology, 142, 158 - 158

Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial

Journal article

MacLaren RE. et al, (2023), Nature Medicine

Choroideremia: The Endpoint Endgame.

Journal article

Abdalla Elsayed MEA. et al, (2023), Int J Mol Sci, 24

Current and Future Landscape in Genetic Therapies for Leber Hereditary Optic Neuropathy.

Journal article

Shamsnajafabadi H. et al, (2023), Cells, 12

Microperimetry as an Outcome Measure in RPGR-associated Retinitis Pigmentosa Clinical Trials

Journal article

Taylor LJ. et al, (2023), Translational Vision Science & Technology, 12, 4 - 4

Choroideremia: Toward Regulatory Approval of Retinal Gene Therapy.

Journal article

Yusuf IH. and MacLaren RE., (2023), Cold Spring Harb Perspect Med, 13

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