Enhanced efficacy and increased long-term toxicity of CNS-directed, AAV-based combination therapy for Krabbe disease

Sequence data from Twitcher (Twi) mice, a model of Krabbe disease (Infantile globoid cell leukodystrophy). Mice were treated with a combination of hematopoietic stem-cell transplantation, a small-molecule substrate reduction therapy (SRT) drug (L-cycloserine), and an AAV2/9 gene therapy regimen. This study contains targeted sequencing data from 82 capture probes that span the entire AAV vector, including the inverted terminal repeats (ITRs), the CAGGS promoter, and the human GALC cDNA. Sequence was generated from hepatic tumors and matched normal-appearing liver tissue from 10 animals.