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The CRISPR/Cas9 ALMS1 -encoding plasmids (KN414632, OriGene) were also applied by lipofectamine transfection according to the manufacturers instructions, and cells were analyzed after puromycin (P8833, Sigma) selection

F.SchroederM

"Spinocerebellar ataxia type-1 and spinobulbar muscular atrophy gene products interact with glyceraldehyde-3-phosphate dehydrogenase"

Free from the acyl group, carnitine can be transported back out of the matrix by CACT and re-used for LCFA shuttling

Duration of hospital stay, including day of hLB-001 infusion, was four days for Participant 1, five days for Participants 2 and 4, and six days for Participant 3