AAV vectors with myelin protein zero promoter and uses thereof for treating SCHWANN cell-associated diseases like CHARCOT-MARIE-TOOTH disease

A method of treating Charcot-Marie-Tooth disease in a subject in need thereof, wherein the method comprises:

• selecting the subject as suffering from Charcot-Marie-Tooth disease;
• administering to said subject a viral vector that comprises a first nucleic acid sequence that can be transcribed into a therapeutic related polynucleotide, and wherein the viral vector is an adeno-associated virus (AAV) vector, wherein the expression of the first therapeutic related polynucleotide is under the control of a promoter selected from:
• a) a full-length myelin protein zero (Mpz) promoter, wherein the full-length Mpz promoter is a full-length rat or full-length human Mpz promoter, wherein the promoter has a sequence with at least 75% sequence homology or sequence identity with SEQ ID NO. 4 or SEQ ID NO. 18, optionally at least 80%, or 82%, or 84%, or 86%, or 88%, or 90%, or 92%, or 94%, or 96%, or 97%, or 98%, or 99%, or 100% sequence identity or sequence homology to SEQ ID NO. 4 or SEQ ID NO. 18;
• or
• b) a minimal myelin protein zero (miniMpZ) promoter, wherein the minimal Mpz promoter is a rat or human minimal Mpz promoter, wherein the promoter has a sequence with at least 75% sequence homology or sequence identity with SEQ ID NO. 5 or SEQ ID NO. 22.