Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Rett syndrome

Methyl CpG binding protein 2 (MECP2; RTT)

Mouse studies suggest MECP2 gene therapy could help treat Rett syndrome. Mutations in MECP2 are the most frequent cause of Rett syndrome. In Mecp2-/- mice, viral delivery of Mecp2 yielded physiologically relevant levels of Mecp2 in the brain and restored normal neuronal soma size, and it increased survival compared with viral delivery of a control gene. In Mecp2-/- female mice, Mecp2 gene transfer decreased motor impairment and seizure frequency compared with transfer of a control gene. Next steps include modifying the viral delivery vector for optimal expression of the gene.

SciBX 6(36); doi:10.1038/scibx.2013.1004
Published online Sept. 19, 2013

Patent and licensing status unavailable

Garg, S.K. et al. J. Neurosci.; published online Aug. 21, 2013;
doi:10.1523/JNEUROSCI.1854-13.2013
Contact: Gail Mandel, Oregon Health & Science University, Portland, Ore.
e-mail:

mandelg@ohsu.edu