Methods and compositions for inhibiting PMP22 expression

#The present application is being filed along with a Sequence Listing in electronic format. The Sequence Listing is provided as a file entitled BIOL0287USC1SEQ_ST25.txt created Aug. 30, 2021, which is 8 kb in size. The information in the electronic format of the sequence listing is incorporated herein by reference in its entirety.

#The present embodiments provide methods, compounds, and compositions useful for inhibiting PMP22 expression, which can be useful for treating, preventing, or ameliorating a disease associated with PMP22.

#Charcot-Marie-Tooth Disease (CMT) is a neurological disorder that affects motor and sensory nerve function. Typical symptoms include muscle weakness in the feet and lower legs, foot deformities, and mild to severe pain. There is no cure for CMT, and treatment typically includes physical therapy, occupational therapy, use of braces, and/or use of pharmaceuticals for pain relief. Several inherited genetic mutations are associated with CMT. For example, duplication of Peripheral Myelin Protein 22 (PMP22) is associated with CMT, specifically CMT Type 1A.

#It is to be understood that both the foregoing general description and the following detailed description are exemplary and explanatory only and are not restrictive. Herein, the use of the term “including” as well as other forms, such as “includes” and “included”, is not limiting.

#The section headings used herein are for organizational purposes only and are not to be construed as limiting the subject matter described. All documents, or portions of documents, cited in this application, including, but not limited to, patents, patent applications, articles, books, treatises, and GenBank and NCBI reference sequence records are hereby expressly incorporated by reference for the portions of the document discussed herein, as well as in their entirety.

#It is understood that the sequence set forth in each sequence ID Number (SEQ ID NO.) herein is independent of any modification to a sugar moiety, an internucleoside linkage, or a nucleobase. As such, oligonucleotides defined by a SEQ ID NO. may comprise, independently, one or more modifications to a sugar moiety, an internucleoside linkage, or a nucleobase.