Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors

Alona O. Barnea-Cramer; Mandeep Singh; Dominik Fischer; Samantha De Silva; Michelle E. McClements; Alun R. Barnard; Robert E. MacLaren

doi:10.1016/j.ymthe.2020.01.023

Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors

Alona O. Barnea-Cramer, Mandeep Singh, Dominik Fischer, Samantha De Silva, Michelle E. McClements, Alun R. Barnard, Robert E. MacLaren

School of Medicine

Research output: Contribution to journal › Article › peer-review

6 Scopus citations

Abstract

Barnea-Cramer et al. show retinal reconstruction and rescue of vision in heritably blind mice following ex vivo minicircle or viral gene correction and cell transplantation of photoreceptor precursors. Converging cell and gene therapies may be applied in future studies toward ex vivo correction of patient-specific cells to replace lost photoreceptors in hereditary neurodegeneration.

Original language	English (US)
Pages (from-to)	830-844
Number of pages	15
Journal	Molecular Therapy
Volume	28
Issue number	3
DOIs	https://doi.org/10.1016/j.ymthe.2020.01.023
State	Published - Mar 4 2020

Keywords

cell delivery
cell therapy
correction
degeneration
gene
gene therapy
photoreceptors
regeneration
retina

ASJC Scopus subject areas

Molecular Medicine
Molecular Biology
Genetics
Pharmacology
Drug Discovery

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10.1016/j.ymthe.2020.01.023

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title = "Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors",

abstract = "Barnea-Cramer et al. show retinal reconstruction and rescue of vision in heritably blind mice following ex vivo minicircle or viral gene correction and cell transplantation of photoreceptor precursors. Converging cell and gene therapies may be applied in future studies toward ex vivo correction of patient-specific cells to replace lost photoreceptors in hereditary neurodegeneration.",

keywords = "cell delivery, cell therapy, correction, degeneration, gene, gene therapy, photoreceptors, regeneration, retina",

author = "Barnea-Cramer, {Alona O.} and Mandeep Singh and Dominik Fischer and {De Silva}, Samantha and McClements, {Michelle E.} and Barnard, {Alun R.} and MacLaren, {Robert E.}",

note = "Funding Information: This study was supported by MRC , the Wellcome Trust , the Royal College of Surgeons of Edinburgh , the Moorfields Eye Hospital Development Fund , the NIHR Oxford Biomedical Research Centre , and The University of Oxford Clarendon Fund . Publisher Copyright: {\textcopyright} 2020 The American Society of Gene and Cell Therapy",

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AU - Barnea-Cramer, Alona O.

AU - Singh, Mandeep

AU - Fischer, Dominik

AU - De Silva, Samantha

AU - McClements, Michelle E.

AU - Barnard, Alun R.

AU - MacLaren, Robert E.

N1 - Funding Information: This study was supported by MRC , the Wellcome Trust , the Royal College of Surgeons of Edinburgh , the Moorfields Eye Hospital Development Fund , the NIHR Oxford Biomedical Research Centre , and The University of Oxford Clarendon Fund . Publisher Copyright: © 2020 The American Society of Gene and Cell Therapy

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AB - Barnea-Cramer et al. show retinal reconstruction and rescue of vision in heritably blind mice following ex vivo minicircle or viral gene correction and cell transplantation of photoreceptor precursors. Converging cell and gene therapies may be applied in future studies toward ex vivo correction of patient-specific cells to replace lost photoreceptors in hereditary neurodegeneration.

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Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors

Abstract

Keywords

ASJC Scopus subject areas

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