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Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

Symptoms are similar to other viral hepatitis and include loss of appetite, fatigue, nausea, vomiting, abdominal pain, joint pain, mild fever, dark urine, and jaundice (yellowing of the skin and eyes due to accumulation of bilirubin secondary to liver dysfunction) (Merck 2007)

Furthermore, as new pharmacological agents enter clinical trials, it is essential to establish robust endpoints that reflect meaningful improvements in joint function and quality of life for patients

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Cui et al., Metabolic regulation of gene expression by histone lactylation