Approach

Summary

Licensing status

Publication and contact information

Disease models

Mouse model for autosomal-dominant leukodystrophy (ADLD)

A mouse model for adult-onset ADLD could be useful for identifying myelin regeneration therapies to treat ADLD and multiple sclerosis (MS). ADLD is a rare genetic disease caused by duplication of lamin B1 (LMNB1) and is often misdiagnosed as chronic progressive MS. Mice engineered to overexpress Lmnb1 in oligodendrocytes showed increased myelin abnormalities, demyelination and motor deficits compared with wild-type mice. Next steps include testing the effect of myelin regeneration therapies in the model.

SciBX 6(22); doi:10.1038/scibx.2013.557
Published online June 6, 2013

Unpatented; licensing status not applicable

Heng, M.Y. et al. J. Clin. Invest.; published online May 15, 2013;
doi:10.1172/JCI66737
Contact: Ying-Hui Fu, University of California, San Francisco, Calif.
e-mail:
ying-hui.fu@ucsf.edu
Contact: Louis J. Ptáček,
same affiliation as above
e-mail:
ljp@ucsf.edu