Gene editing for controlled expression of episomal genes
AssigneeSpark Therapeutics, Inc.
InventorsSean Armour, Ali Nahvi, Federico Mingozzi, Daniel Cohen, Yahui Lan
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AssigneeSpark Therapeutics, Inc.
InventorsSean Armour, Ali Nahvi, Federico Mingozzi, Daniel Cohen, Yahui Lan
Estimating the term…
Disclosed herein are methods for regulating expression of a gene located on an episomal vector in a subject in need thereof. In particular, methods include administering to the subject one or more gene editing agents that modify a region of the gene to thereby regulate the expression of the gene. Also disclosed are methods of administering to the subject a base editor system that effects a base alteration in a region of the gene or a region of an mRNA transcript of the gene to thereby regulate the expression of the gene.
A method of regulating expression of a gene located on an episomal vector in a subject in need thereof, comprising administering to the subject an editing agent that effects an alteration in a region of an mRNA transcript of the gene to thereby regulate the expression of the gene.
Dependencies are inferred from the claim text.