AAV9-MBGAL
AAV9-MBGAL has been studied in 1 paper since 2015: none in people yet, 1 in animals. The research so far is in animals. The newest paper, May 10, 2015: “Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan”.
1 paper studies it3 findingsUpdated
None of its findings points the other way.
- 1 lab in 1 country
- Funders: Eunice Kennedy Shriver National Institute of Child Health and Human Development and 4 more
- Animals1
What did studies of AAV9-MBGAL find?
3Studies of AAV9-MBGAL measured 3 outcomes; the most studied are motor function (1 study), GM1 ganglioside (1) and lifespan (1).
Motor function
upin mice1
1 study
Motor function
upin mice1
AAV9-MBGAL improves motor function in mice.
“AAV9 treatment resulted in improved performance in multiple tests of motor function and behavior”
Animalsin adult GM1 mice (βGal(-/-))
Human molecular genetics
GM1 ganglioside
downin mice1
1 study
GM1 ganglioside
downin mice1
AAV9-MBGAL decreases GM1 ganglioside in mice (36-76% reduction).
“36-76% reduction in GM1-ganglioside content in the brain”
Animalsin adult GM1 mice (βGal(-/-))brain
Human molecular genetics
Lifespan
upin mice1
1 study
Lifespan
upin mice1
AAV9-MBGAL extends lifespan in mice (316-576 days vs 250-264 days).
“median survival of AAV9 treatment groups (316-576 days) was significantly increased over controls (250-264 days)”
Animalsin adult GM1 mice (βGal(-/-))1 × 10(11) or 3 × 10(11) vg
Human molecular genetics