Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A

Inclusion Criteria: * Subjects 18- 35 years old inclusive with CMT1A will be enrolled * Must exhibit a 1.5 Mb duplication at 17p11.2 inclusive of the peripheral myelin protein 22 (PMP22) gene * Males and females of any ethnic or racial group * Must exhibit weakness of the ankle dorsiflexion muscle (but has full ROM against gravity and is able to stand on heels 3 seconds or greater) * Abnormal nerve conduction velocities * Ability to cooperate for clinical evaluation and repeat nerve conduction studies * Willingness of sexually active subjects to practice a reliable method of contraception during the study Exclusion Criteria: * Active viral infection based on clinical observations or serological evidence of HIV, or Hepatitis B or C infection, herpesvirus or adenovirus * Ongoing immunosuppressive therapy or immunosuppressive therapy within 6 months of starting the trial (e.g., corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin) * Persistent leukopenia or leukocytosis (WBC ≤ 3.5 K/µL or ≥ 20.0 K/µL) or an absolute neutrophil count \< 1.5K/µL * Subjects with AAV1 binding antibody titers ≥ 1:50 as determined by ELISA immunoassay * Subjects with circulating anti-NT-3 titers ≥ 1:50 as determined by ELISA immunoassay * Treat with any investigational medication within 30 days before the infusion of study drug * Abnormal laboratory values considered clinically significant (GGT \> 3XULN, bilirubin ≥ 3.0 mg/dL, creatinine ≥ 1.8 mg/dL,…