Mutations in ATP binding cassette subfamily D member 1 (ABCD1) lead to the loss of adrenoleukodystrophy (ALD) protein function, resulting in X-linked adrenoleukodystrophy, Florian Eichler et al. [122] transduced autologous CD34+ cells from patients with LV vectors and observed the expression of ALD protein in all patients, with majority patients surviving and experiencing few adverse reactions. The gene discussed is ABCD1; the disease is X-linked adrenoleukodystrophy.