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Accession: PRJNA739996 ID: 739996

Base editing rescues the function and survival of cone in a LCA mouse model

We show that base editing can rescue the function and survival of cone photoreceptors in a mouse model of inherited retinal degeneration, which has been a major challenge in the treatment of inherited blindness.
AccessionPRJNA739996
Data TypeRaw sequence reads
ScopeMultispecies
Grants
  • "Medical Scientist Training Program" (Grant ID T32 GM007250, National Institute of General Medical Sciences)
  • "Pre-doctoral Training Program in Molecular Therapeutics" (Grant ID T32 GM008803, National Institute of General Medical Sciences)
  • "CRISPR/Cas9-Mediated Correction of Abca4 Mutations in a Stargardt Mouse Model" (Grant ID F30 EY029136, National Eye Institute)
  • "Retinoids in Vision" (Grant ID R01 EY009339, National Eye Institute)
  • "Use of systems pharmacology to prevent rod and cone photoreceptor degeneration" (Grant ID R24 EY027283, National Eye Institute)
  • "Cole Eye Institute Vision Science Training Program" (Grant ID T32 EY024236, National Eye Institute)
SubmissionRegistration date: 22-Jun-2021
University of California, Irvine
RelevanceMedical
Project Data:
Resource NameNumber
of Links
Sequence data
SRA Experiments33
Other datasets
BioSample4
SRA Data Details
ParameterValue
Data volume, Gbases195
Data volume, Mbytes60240

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