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

Research output: Contribution to journalArticlepeer-review

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 languageEnglish (US)
Pages (from-to)830-844
Number of pages15
JournalMolecular Therapy
Volume28
Issue number3
DOIs
StatePublished - 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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