Intrathecal delivery of recombinant adeno-associated virus encoding Methyl-CpG binding protein 2
View Patent ↗Methods and materials for intrathecal delivery of recombinant Adeno-associated virus 9 (rAAV9) encoding Methyl-CpG binding protein 2 (MECP2) are provided. Use of the methods and materials is contemplated, for example, for the treatment of Rett syndrome.
1. A method of treating Rett syndrome in a patient comprising the step of intrathecal administration of a recombinant adeno-associated virus 9 (rAAV9) encoding Methyl-CpG binding protein 2 (MECP2) to a patient in need thereof, wherein the rAAV9 comprises a self-complementary genome encoding MECP2B and wherein the sequence of the self-complementary genome is set out in SEQ ID NO: 1.
2. The method of claim 1 further comprising the intrathecal administration of iohexol, iobitridol, iomeprol, iopamidol, iopentol, iopromide, ioversol or ioxilan, or mixtures of two or more thereof.
3. The method of claim 1 , further comprising putting the patient in the Trendelenberg position.