Pre-Clinical Assessment of the Proteasomal Inhibitor Bortezomib as a Generalized Therapeutic Approach for Recessively Inherited Disorders
The number of known monogenic rare diseases (~7000) exceeds the number of effective treatments (~500) by more than an order of magnitude underlining the pressing need for generalizable therapeutic approaches for this class of conditions. In this regard, the majority of recessive and x-linked recessi...
| Main Author: | |
|---|---|
| Other Authors: | |
| Language: | en |
| Published: |
Université d'Ottawa / University of Ottawa
2017
|
| Subjects: | |
| Online Access: | http://hdl.handle.net/10393/36066 http://dx.doi.org/10.20381/ruor-20346 |