IT and IV Lentiviral Gene Therapy for X-ALD

This is a Phase I/II clinical trial of gene therapy for treating X-linked adrenoleukodystrophy using a high-safety, high-efficiency, self-inactivating lentiviral vector (LV) TYF-ABCD1 to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the intrathecal and intravenous lentiviral gene transfer clinical protocol.

Inclusion Criteria: 1. X-ALD patients ≥ 1 year of age 2. ALD diagnosis of the brain: evaluation of the VLCFA value in plasma 3. Central imaging of the MRI to examine the damage on the CNS. 4. Neurological function score (NFS) ≥ 1 5. Parent / guardian / patient signing informed consent 6. Patients and their families have a strong willingness to participate in clinical trials, and are willing to bear all the consequences caused by the failure of the trial, and sign an informed consent form Exclusion Criteria: 1. HIV positive patients 2. Stablized condition after statins, Lorenzo's oil, or diet to reduce VLCFA levels 3. Patients who are experiencing severe viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency 4. Cannot perform an MRI 5. Infection or dermatosis at pre-injection site