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1. (WO2018136434) COMPOSITIONS AND METHODS FOR DIAGNOSING AND TREATING PEROXISOMAL DISEASES

Pub. No.:    WO/2018/136434    International Application No.:    PCT/US2018/013908
Publication Date: Fri Jul 27 01:59:59 CEST 2018 International Filing Date: Wed Jan 17 00:59:59 CET 2018
IPC: C12Q 1/00
G01N 33/50
G01N 33/68
Applicants: CHILDREN'S MEDICAL CENTER CORPORATION
OSPEDALE SAN RAFFAELE S.R.L.
Inventors: BIFFI, Alessandra
CAVALCA, Eleonora
Title: COMPOSITIONS AND METHODS FOR DIAGNOSING AND TREATING PEROXISOMAL DISEASES
Abstract:
The invention features compositions and methods for the treatment and prevention of peroxisomal diseases (e.g., neonatal adrenoleukodystrophy and Zellweger syndrome), including in a subject selected as having increased levels of metallothionein polypeptides. The invention also provides compositions and methods for identifying a subject having a peroxisomal disease involving detecting metallothionein polypeptides or polynucleotdies.