Table 1 Study design in rd10/rd10 mice.

From: Preclinical safety and biodistribution of SPVN06, a novel gene- and mutation-independent gene therapy for rod-cone dystrophies

Strain

Treatment

Dose

 

Number and gender

  

Injection volume (µl)

Total dose (vg/eye)

 

WT C57BL/6Ja

No treatment

N/A

0

5 M, 5 F

rd10/rd10

Vehicle (OU)

1

0

7 M, 7 F

 

SPVN06 (OU)

1

1.0 × 108

6 M, 6 F

  1. aData from a separate study performed in the same animal facility in WT mice were included to provide an additional reference point.
  2. All injections were OU; both eyes were treated and received one subretinal administration of either vehicle or SPVN06. The SPVN06 dose of 1.0 × 108 vg/eye was selected based on previous studies conducted with either SPVN06 or its prototypes at various doses.
  3. F female, M male, N/A not applicable, OU oculus uterque, vg vector genomes, WT wild-type.
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