Researchers Submit Patent Application, "Materials And Methods For Treatment Of Amyotrophic Lateral Sclerosis", for Approval (USPTO 20230392134).
In: Gene Therapy Weekly, 2023-12-25, S. 3985-3985
serialPeriodical
Zugriff:
This document is a patent application for a method of treating amyotrophic lateral sclerosis (ALS) by editing the C9ORF72 gene in human cells. The method involves delivering CRISPR systems, including guide ribonucleic acids (gRNAs) and site-directed deoxyribonucleic acid (DNA) endonucleases, to the cells. The endonucleases, such as Cas9, cause double-stranded breaks (DSBs) within specific regions of the C9ORF72 gene, resulting in the permanent deletion of the hexanucleotide repeat associated with ALS. The patent application provides various embodiments and details of the method, including the use of different gRNAs, delivery methods, and modifications of the endonucleases. [Extracted from the article]
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Titel: |
Researchers Submit Patent Application, "Materials And Methods For Treatment Of Amyotrophic Lateral Sclerosis", for Approval (USPTO 20230392134).
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Zeitschrift: | Gene Therapy Weekly, 2023-12-25, S. 3985-3985 |
Veröffentlichung: | 2023 |
Medientyp: | serialPeriodical |
ISSN: | 1078-2842 (print) |
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