TABLE 1
FROM:
Complete Correction of Enzymatic Deficiency and Neurochemistry in the GM1-gangliosidosis Mouse Brain by Neonatal Adeno-associated Virus–mediated Gene Delivery
M L D Broekman, R C Baek, L A Comer, J L Fernandez, T N Seyfried and M Sena-Esteves
BACK TO ARTICLETable 1. Effect of AAV vector on the concentration of lipids in the brain of adult
gal mice
| Strain | Treatment | N a | Total gangliosides | GM1 b | GD1a b | GT1a b | GD1b b | GT1b b | GQ1b b | GA1 | Cholesterol c | Cerebrosides c | Sulfatides c |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| C57BL/6 | — | 3 | 412 17 | 53 1 | 164 4 | 14 1 | 29 1 | 118 8 | 34 4 | Not detectable | 6.9 0.3 | 2.7 0.1 | 0.81 0.14 |
gal-/-
| — | 3 | 824 65d
| 476 42d
| 153 12 | 16 1 | 40 1d
| 110 7 | 30 4 | 1.64 0.05 | 6.6 0.3 | 1.6 0.2e
| 0.31 0.04e
|
gal-/-
| AAV | 7 | 444 21 | 47 2 | 169 9 | 17 1 | 32 1 | 136 6 | 43 3 | Not detectable | 7.4 0.4 | 3.0 0.3 | 0.82 0.09 |
AAV, adeno-associated virus.
a N, the number of independent mice analyzed.
b Ganglioside concentrations were expressed as microgram sialic acid/100 mg dry weight. Concentration of GA1 was expressed as mg/100 mg dry weight. Values represent the mean
1SEM. Gangliosides were identified according to the nomenclature system of Svennerholm.43 Gangliosides that comprised less than 1% of the total ganglioside content were not included (GM2, GM3, and GM4). Determined from densitometric scanning of HPTLC plates as shown in Figure 3a and b.
c Lipid concentrations expressed as mg/100 mg dry weight. Values represent the mean
1SEM. Determined from densitometric scanning of HPTLC plates as shown in Figure 6.
d Indicates that the concentration is significantly different from that in wild-type C57BL/6 and AAV-treated
gal-/- mice at P<0.01 by one-way ANOVA.
e Indicates that the concentration is significantly different from that in wild-type C57BL/6 controls and AAV-treated
gal-/- mice at P<0.05 by one-way ANOVA.
