IP Library › Granted Patent US 12702720
Granted Patent B2
US 12702720 · App. 17/924,469 · Granted Aug 11, 2026

Genomic insulator element exhibiting enhancer blocking activities in lymphocytes and uses thereof

Inventors: Sheng Zhou (Memphis, TN); Brian Sorrentino (Memphis, TN); Yong Cheng (Memphis, TN)
Assignee: St. Jude Children's Research Hospital, Inc.
A61K48/0008C12N15/86C12N2740/15043C12N2830/40C12N2830/48
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Quick Facts
Patent No.
US 12702720
App. No.
17/924,469
Granted
Aug 11, 2026
Kind
B2
Abstract

A construct including a genomic insulator element that exhibits strong enhancer blocking activities in T lymphocytes is provided as are host cells, pharmaceutical compositions and methods of using the construct in the treatment of disease, in particular a disease to be treated with a retroviral vector-modified T lymphocyte.

Claims (11)

1 . A construct comprising at least one copy of a genomic insulator element having a core sequence selected from the group of CACTGCCCTCCAGTGGCCA (SEQ ID NO:1), CAGCGCCACCTGCAGGCCA (SEQ ID NO:2), CTTCCAGCAGGAGGAGGCA (SEQ ID NO:3), TGGCCGCTAGAGGGCACGC (SEQ ID NO: 4), AAGCACCATCTACTGGTCT (SEQ ID NO:5), CTGCCGCCAGATGGCGCTC (SEQ ID NO: 6), TCAGCACTAGATGGCACCC (SEQ ID NO:7), GAGTGACACCTAGTGGCCC (SEQ ID NO:8), CAGCGCCATCTGGGGGCCG (SEQ ID NO: 9), TCGCCAGTAGGGGGCGCAA (SEQ ID NO:10), TGCTGCCCCCTGGTGGCCA (SEQ ID NO: 11), TGCTGCTCCCTTATGGCCA (SEQ ID NO:12), AGGCCACCAGATGGCATTG (SEQ ID NO: 13), CTGCCACGAGGGGGCGGCA (SEQ ID NO: 14), TTGCGCCCCCTGCTGGCGA (SEQ ID NO:15), CGTCGCCACCTTCTGGTAA (SEQ ID NO: 16), CAGTGCCCTCTGGTGGTAG (SEQ ID NO: 17), TTATGCCCCCTGCAGGACA (SEQ ID NO:18), CGCCCAGAAGGTGGCGGCA (SEQ ID NO:19), and CACTGCCCCCTAGTGGACC (SEQ ID NO:20), wherein the construct is a viral vector, transgene cassette, or transposon system, wherein the genomic insulator element comprises a sequence selected from the group of SEQ ID NO: 21, SEQ ID NO: 22, SEQ ID NO:23, SEQ ID NO:24, SEQ ID NO: 25, SEQ ID NO: 26, SEQ ID NO:27, SEQ ID NO:28, SEQ ID NO:29, SEQ ID NO: 30, SEQ ID NO: 31, SEQ ID NO: 32, SEQ ID NO: 33, SEQ ID NO: 34, SEQ ID NO: 35, SEQ ID NO: 36, SEQ ID NO: 37, SEQ ID NO: 38, SEQ ID NO:39 and SEQ ID NO: 40.

2 . The construct of claim 1 , wherein the viral vector is a retroviral vector.

3 . The construct of claim 2 , wherein the retroviral vector is a lentiviral vector.

4 . The construct of claim 1 , wherein the construct is a gene therapy construct.

5 . The construct of claim 1 further comprising a therapeutic agent, optionally wherein the therapeutic agent comprises a gene of interest a protein, a dominant negative mutant, an RNA interference agent, or an miRNA.

6 . A host cell comprising the construct of claim 1 .

7 . The host cell of claim 6 , wherein said host cell is a lymphocyte.

8 . A pharmaceutical composition comprising the construct of claim 1 and a pharmaceutically acceptable carrier.

9 . A method for treating a disease comprising administering a construct of claim 5 , wherein the therapeutic agent mediates treatment of the disease.

10 . The method of claim 9 , wherein the therapeutic agent comprises a protein, a dominant negative mutant, an RNA interference agent, or an miRNA.

11 . The method of claim 9 , wherein the disease is a primary immunodeficiency, haemoglobinopathy or inborn error of metabolism.