The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Nov. 26, 2024
Filed:
Nov. 30, 2022
Eberhard-karls-universität Tübingen Universitätsklinikum, Tübingen, DE;
Justin Antony Selvaraj, Weil am Rhein, DE;
Markus Mezger, Tübingen, DE;
Eberhard-Karls-Universität Tübingen Universitätsklinikum, Tübingen, DE;
Abstract
The present invention relates to a gene editing system for editing the arylsulfatase A (ARSA) gene, polynucleotides, sgRNA molecules and repair templates which may form parts of the gene editing system, vectors and host cells incorporating said nucleic acid molecules, a pharmaceutical composition, and kits. It also relates to a method of gene editing the arylsulfatase A (ARSA) gene in a cell or a subject, and to a method of treating a disease or disorder associated with functional deficiency of the ARSA enzyme in a subject.