Methods for screening cereblon modifying compounds

Abstract

A method of screening a cereblon modifying compound for treating a disease or disorder, comprising: obtaining a sample; determining a first protein level of SALL4; administering the cereblon modifyingcompound to the sample; determining a second protein level of SALL4; comparing the first level and the second protein level of SALL4 to determine if the cereblon modifying compound induces degradation of SALL4; and selecting the cereblon modifying compound that does not induce degradation of SALL4.

Description

[0001] Cross References to Related Applications [0002] This application claims the benefit of priority to U.S. Provisional Patent Application No. 62 / 617,112, filed January 12, 2018, and U.S. Provisional Patent Application No. 62 / 710,401, filed February 16, 2018, the disclosure of each Provisional Patent Application The contents are incorporated herein by reference in their entirety. [0003] sequence listing [0004] This application incorporates by reference the Sequence Listing, which is filed with this application as a text file entitled 14247-269-228_SEQ_LISTING.txt, created on January 8, 2019 and is 57,330 bytes in size . [0005] 1. Technical field [0006] In certain embodiments, provided herein are methods for screening for cerebellar protein-modifying compounds that have a reduced risk of inducing teratogenicity. In certain embodiments, methods for determining whether a cerebellin-modifying compound induces a teratogenic effect are also provided. [0007] 2. Back...

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Problems solved by technology

Its defective activity causes repair defects in patients with xeroderma pigmentosa complementation group E (XPE)

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