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. 2015 Apr;4(2):139–163. doi: 10.3978/j.issn.2224-4336.2015.04.03

Table 5. Examples of therapies applied in the retinal dystrophies.

Approach   Context   Strategy summary Studies
Gene therapy   Human clinical trial   RPE65 delivered by Adeno-associated Virus (AAV) (263)
  Mouse model   CHM delivered by Adeno-associated Virus (AAV) (264)
  Human clinical trial   CHM delivered by Adeno-associated Virus (AAV) (270)
  In vitro model   CEP290 delivered by Lentivirus vector (271)
  Rat model   MERTK delivered by Adeno-associated Virus (AAV) (272)
  Mouse model   CRB1 and CRB2 delivered by Adeno-associated Virus (AAV) (273)
Cell replacement   Rat model   Human RPC transplantation restores mature rat retina cells (274)
  Mouse model   Adult mouse iPSC differentiate into photoreceptors (267)
  Mouse model   RPCs differentiate and integrate into functional photoreceptors (269)
  Mouse model   RPCs differentiate into photoreceptors (268)
Retinal implants   Clinical trials   Argus II Retinal Prosthesis System (260,261)