IP Library Granted Patent US 11,352,646
Granted Patent B2
US 11,352,646 · App. 17/289,653 · Granted Jun 7, 2022

Vector system for expressing regulatory RNA

Inventors: Tyler Lahusen (Rockville, MD); Lingzhi Xiao (Rockville, MD); Charles David Pauza (Rockville, MD)
Assignee: American Gene Technologies International Inc.
C12N15/86C12N15/113C12N2310/14C12N2740/15023C12N2740/15043
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Quick Facts
Patent No.
US 11,352,646
App. No.
17/289,653
Granted
Jun 7, 2022
Kind
B2
Abstract

Viral vectors, lentiviral particles, and modified cells are disclosed. They encode or express a small RNA capable of targeting the KIF11 gene. In embodiments, the viral vectors and lenti viral particles further comprise and a KIF11 gene whose non-coding region has been modified such that it is resistant to activity by the small RNA.

Claims (19)

1. A modified mesenchymal stem cell comprising a mesenchymal stem cell infected with a lentiviral particle, wherein the lentiviral particle comprises:

an envelope protein capable of infecting the mesenchymal stem cell; and

a first nucleotide sequence encoding a small RNA capable of binding a non-coding region of a host copy of KIF11,

wherein the small RNA comprises a sequence having at least 80% sequence identity with at least one of SEQ ID NO: 1, SEQ ID NO: 2, or SEQ ID NO: 3.

2. The modified mesenchymal stem cell of claim 1 , wherein the small RNA comprises at least one of SEQ ID NO: 1, SEQ ID NO: 2, or SEQ ID NO: 3.

3. A method of producing a modified mesenchymal stem cell, the method comprising:

infecting a mesenchymal stem cell with an effective amount of a lentiviral particle, wherein the lentiviral particle comprises:

an envelope protein capable of infecting the mesenchymal stem cell; and

a first nucleotide sequence encoding a small RNA capable of binding a non-coding region of a host copy of KIF11,

wherein the small RNA comprises a sequence having at least 80% sequence identity with at least one of SEQ ID NO: 1, SEQ ID NO: 2, or SEQ ID NO: 3.

4. The method of claim 3 , wherein the small RNA comprises at least one of SEQ ID NO: 1, SEQ ID NO: 2, or SEQ ID NO: 3.

5. A viral vector comprising a first nucleotide sequence encoding a small RNA capable of binding a non-coding region of a host copy of KIF11,

wherein the small RNA comprises a sequence having at least 80% sequence identity with at least one of SEQ ID NO: 1, SEQ ID NO: 2, or SEQ ID NO: 3.

6. The viral vector of claim 5 , wherein the small RNA comprises at least one of SEQ ID NO: 1, SEQ ID NO: 2, or SEQ ID NO: 3.

7. A lentiviral particle produced by a packaging cell and capable of infecting a target cell, the lentiviral particle comprising:

an envelope protein capable of infecting the target cell; and

a first nucleotide sequence encoding a small RNA capable of binding a non-coding region of a host copy of KIF11,

wherein the small RNA comprises a sequence having at least 80% sequence identity with at least one of SEQ ID NO: 1, SEQ ID NO: 2, or SEQ ID NO: 3.

8. The lentiviral particle of claim 7 , wherein the small RNA comprises at least one of SEQ ID NO: 1, SEQ ID NO: 2, or SEQ ID NO: 3.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 16, 2022
From: LAHUSEN, TYLER; XIAO, LINGZHI; PAUZA, CHARLES DAVID
To: AMERICAN GENE TECHNOLOGIES INTERNATIONAL INC.
Reel/Frame 059287/0236 →
Continuity (2)
Provisional Application 62755985 · Nov 5, 2018
Related Publication 20210403949A1 · Dec 30, 2021
Cited By (1)
US 12,709,753