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A new PDE6A missense variant p.Arg544Gln in rod-cone dystrophy.

Takaaki HayashiKei MizobuchiShuhei KameyaKazutoshi YoshitakeTakeshi IwataTadashi Nakano
Published in: Documenta ophthalmologica. Advances in ophthalmology (2021)
This is the second case of a patient with AR-RCD associated with PDE6A in the Japanese population. These findings will contribute to a better clinical understanding of PDE6A-associated RCD and valuable insights for gene therapy trials.
Keyphrases
  • gene therapy
  • case report
  • early onset
  • intellectual disability