Patient visual behavior following treatment with rAAV2/8.hRKp.AIPL1

  • Children with AIPL1 deficiency typically have only light perception vision and so are unable to perform any vision-guided tasks. After treatment, the child performs vision guided tasks
  • This improvement will transform their life, given that vision impacts many areas of development; children who are profoundly visually impaired often suffer other negative implications on their behavioral, emotional and communication development

Patient 2, female, left eye (OS), c.834G>A p.(Trp278Ter) hom
Age at time of treatment: 2y 10m

Patient 8, male, left eye (OS), c.834G>A p.(Trp278Ter) hom
Age at time of treatment: 3y 2m