	US 12,360,123 B2
	E3 ubiquitin ligase (UBE3A) protein targets
Veronica Costa, Basel (CH); Thomas Peter John Dunkley, Basel (CH); Nikhil Janak Pandya, Basel (CH); and Ravi Jagasia, Basel (CH)
Assigned to Hoffmann-La Roche Inc., Little Falls, NJ (US)
Appl. No. 17/423,359
Filed by Hoffmann-La Roche Inc., Little Falls, NJ (US)
PCT Filed Jan. 15, 2020, PCT No. PCT/EP2020/050861 § 371(c)(1), (2) Date Jul. 15, 2021, PCT Pub. No. WO2020/148310, PCT Pub. Date Jul. 23, 2020.
Claims priority of application No. 19152337 (EP), filed on Jan. 17, 2019.
Prior Publication US 2022/0091137 A1, Mar. 24, 2022
Int. Cl. G01N 33/68 (2006.01); C12Q 1/25 (2006.01)

CPC G01N 33/6896 (2013.01) [C12Q 1/25 (2013.01); G01N 2333/9015 (2013.01); G01N 2500/02 (2013.01); G01N 2800/60 (2013.01)]

15 Claims

1. A method for measuring UBE3A protein expression modulation in a cerebral spinal fluid (CSF) sample comprising the steps:

a) providing a CSF sample of an animal which has been treated with a UBE3A modulator comprising an antisense oligonucleotide,

b) measuring a protein expression level in the sample of step a) of at least one protein selected from the group consisting of: CCDC88A, DST, FAM127A, FAM127B, FAM127C, PEG10, TCAF1, and PPID,

c) comparing the protein expression level of the at least one protein measured in step b) to the protein expression level of the at least one protein in a control, wherein a modulated protein expression level of the at least one protein measured in step b) compared to the protein expression level of the at least one protein in the control is indicative for UBE3A protein expression modulation.