Molecular Therapy

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

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Table 1. Effect of AAV vector on the concentration of lipids in the brain of adult bold italic betagal 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/63412plusminus1753plusminus1164plusminus414plusminus129plusminus1118plusminus834plusminus4Not detectable6.9plusminus0.32.7plusminus0.10.81plusminus0.14
betagal-/- 3824plusminus65d 476plusminus42d 153plusminus1216plusminus140plusminus1d 110plusminus730plusminus41.64plusminus0.056.6plusminus0.31.6plusminus0.2e 0.31plusminus0.04e
betagal-/- AAV7444plusminus2147plusminus2169plusminus917plusminus132plusminus1136plusminus643plusminus3Not detectable7.4plusminus0.43.0plusminus0.30.82plusminus0.09

 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 meanplusminus1SEM. 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 meanplusminus1SEM. 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 betagal-/- 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 betagal-/- mice at P<0.05 by one-way ANOVA.

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