2026
mRNA delivery to the retina restores REP1 function in choroideremia
L. Lemos# ∙ Pedro Antas#, C. Carvalho, M. Castro, D. Bruno, A.F. Fonseca, S. Rakshit, M.C. Seabra. #Equal contribution.
Molecular Therapy Advances (2026). Open access
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2024
Modeling choroideremia disease with isogenic induced pluripotent stem cells
A.F. Fonseca, R. Coelho, M. Lopes- da-Silva, L. Lemos, M.J. Hall, D. Oliveira, A.S. Falcão, S.T., M.C. Seabra, P. Antas
Stem Cells and Development (2024). Open access
Toward low-cost gene therapy: mRNA-based therapeutics for treatment of inherited retinal diseases
P. Antas#, C. Carvalho#, J. Cabral-Teixeira, L. Lemos, M.C. Seabra. #Equal contribution.
Trends in Molecular Medicine (2024). Open access
2023
Gene therapy for inherited retinal diseases: exploiting new tools in genome editing and nanotechnology
C. Carvalho, L. Lemos, P. Antas, M.C. Seabra
Frontiers in Ophthalmology (2023). Open access
2018
Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia
K. Xue, J.K. Jolly, A.R. Barnard, A. Rudenko, A.P. Salvetti, M.I. Patrício, T.L. Edwards, M. Groppe, H.O. Orlans, T. Tolmachova, G.C. Black, A.R. Webster, A.J. Lotery, G.E. Holder, S.M. Downes, M.C. Seabra, R.E. MacLaren
Nature Medicine (2018)
2016
Visual acuity after retinal gene therapy for choroideremia
T.L. Edwards, J.K. Jolly, M. Groppe, A.R. Barnard, C.L. Cottriall, T. Tolmachova, G.C. Black, A.R. Webster, A.J. Lotery, G.E. Holder, K. Xue, S.M. Downes, M.P. Simunovic, M.C. Seabra, R.E. MacLaren
The New England Journal of Medicine (2016)
2014
Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial
R.E. MacLaren, M. Groppe, A.R. Barnard, C.L. Cottriall, T. Tolmachova, L. Seymour, K.R. Clark, M.J. During, F.P. Cremers, G.C. Black, A.J. Lotery, S.M. Downes, A.R. Webster, M.C. Seabra
The Lancet (2014)
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