Scaav-gusb

1 paper3 findings

1 lab in 1 country

Funders: National Eye Institute and 2 more

Animals1

Outcomes

3

Stroma thickness

downin mice1

1 study

Scaav-gusb decreases stroma thickness in mice.
“Both corneal cloudiness and stroma thickness decreased”
AnimalsscAAV-Gusbcornea4-12 weeks
Self-complementary AAV vector therapy for treating corneal cloudiness of mucopolysaccharidosis type VII (MPS VII)
The ocular surface12 Jan 2024

Corneal cloudiness

downin mice1

1 study

Scaav-gusb decreases corneal cloudiness in mice.
“Both corneal cloudiness and stroma thickness decreased, and there was the presence of β-Glu enzyme activity in the Gusb corneas receiving scAAV-Gusb virus”
AnimalsscAAV-Gusbcornea4-12 weeks
Self-complementary AAV vector therapy for treating corneal cloudiness of mucopolysaccharidosis type VII (MPS VII)
The ocular surface12 Jan 2024

Lifespan

upin mice1

1 study

Scaav-gusb extends lifespan in mice (median survival from 22.5 to 50 weeks).
“The treatment improves the survival rate of Gusb mice, prolonging the median survival rate from 22.5 weeks (untreated) to 50 weeks (treated).”
AnimalsscAAV-Gusb
Self-complementary AAV vector therapy for treating corneal cloudiness of mucopolysaccharidosis type VII (MPS VII)
The ocular surface12 Jan 2024

Latest

The Ocular Surface12 Jan

Self-complementary AAV vector therapy for treating corneal cloudiness of mucopolysaccharidosis type VII (MPS VII)

Experiments in miceInterventions