IP Library Granted Patent US 12,331,086
Granted Patent B2
US 12,331,086 · App. 16/349,672 · Granted Jun 17, 2025

Artificially engineered SC function control system

Inventors: Seok Joong Kim (Seoul, KR); Dong Woo Song (Seoul, KR); Young Bin Hong (Seoul, KR); Byung Ok Choi (Seoul, KR); Jae Young Lee (Seoul, KR); Jung Min Lee (Seoul, KR)
Assignee: TOOLGEN INCORPORATED
C07K14/315A61K35/30C12N15/113C12N15/86C12N2310/10C12N2310/20
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Quick Facts
Patent No.
US 12,331,086
App. No.
16/349,672
Granted
Jun 17, 2025
Kind
B2
Abstract

The present invention relates to an artificially manipulated SC function-controlling factor for SC function control and/or the treatment or alleviation of a disease due to an SC function disorder, and to a use thereof. More specifically, the present invention relates to a system capable of performing artificial SC function control and/or treating or alleviating a disease due to an SC function disorder, the system comprising: an artificially manipulated SC function-controlling factor for SC function control and/or the treatment or alleviation of a disease due to an SC function disorder; and/or a composition for treating or alleviating a disease due to an SC function disorder. In a specific aspect, the present invention relates to an SC function-controlling system by an SC function-controlling factor, such as artificially manipulated PMP22, and/or an expression product thereof.

Claims (8)

1. A composition for editing a PMP22 gene in a cell, comprising:

a Cas9 protein derived from Streptococcus pyogenes or a nucleic acid encoding the Cas9 protein; and

a guide RNA comprising a crRNA and a tracrRNA or a nucleic acid encoding the guide RNA,

wherein the guide RNA targets a sequence of SEQ ID NOs: 14 to 17, which is a TATA-box site of a human PMP22 gene.

2. The composition of claim 1 , wherein the guide RNA is in the form of chimeric single stranded RNA (sgRNA).

3. The composition according to claim 2 , wherein the composition comprises the Cas9 protein and the guide RNA, in which the Cas9 protein are complexed with the guide RNA as a form of ribonucleoprotein (RNP).

4. The composition according to claim 2 , wherein the composition is in the form of one or more vectors.

5. The composition according to claim 4 , wherein the vector is a viral or non-viral vector, wherein the non-viral vector is plasmids, and wherein the viral vector is selected from a group consisting of a retrovirus, a lentivirus, an adenovirus, adeno-associated virus (AAV), vaccinia virus, a poxvirus and a herpes simplex virus.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded May 14, 2019
From: KIM, SEOK JOONG; SONG, DONG WOO; HONG, YOUNG BIN; CHOI, BYUNG OK; LEE, JAE YOUNG; LEE, JUNG MIN
To: TOOLGEN INCORPORATED
Reel/Frame 049168/0262 →
Continuity (2)
Provisional Application 62421516 · Nov 14, 2016
Related Publication 20190276503A1 · Sep 12, 2019
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