Engineered itr sequences and methods of use
AssigneeBioverativ Therapeutics Inc.
InventorsAjay Maghodia, Philip Zakas
Life of this patent
Estimating the term…
Abstract
The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a target sequence. In some embodiments, the first ITR and/or the second ITR is an ITR of a non-adeno-associated virus (AAV). Also disclosed are methods of using the nucleic acid molecules in gene therapy applications.
Claims29
A nucleic acid molecule comprising a first inverted terminal repeat (ITR) and a second ITR flanking a genetic cassette comprising a heterologous polynucleotide sequence, wherein:
- the first ITR comprises a polynucleotide sequence at least about 75% identical to:
- nucleotides 1-49, 50-58, and 59-125 of SEQ ID NO:1,
- nucleotides 1-27 and 50-114 of SEQ ID NO:15, or
- SEQ ID NO:
- 25;
- and
- the second ITR comprises a polynucleotide sequence at least about 75% identical to:
- nucleotides 1-67, 68-76, and 77-125 of SEQ ID NO:2,
- nucleotides 1-65 and 88-114 of SEQ ID NO:16, or
- SEQ ID NO:
- 26.
(canceled)
5-13. (canceled)
(canceled)
17-25. (canceled)
A nucleic acid molecule comprising a first inverted terminal repeat (ITR) and a second ITR flanking a genetic cassette comprising a heterologous polynucleotide sequence,
- wherein the first ITR comprises SEQ ID NOs:1, 3, 5, 9, 13, 15, 17, 19, 21, 23, or 25 and the second ITR comprises SEQ ID NOs:2, 4, 6, 10, 14, 16, 18, 20, 22, 24, or 26.
28-43. (canceled)
(canceled)
47-56. (canceled)
58-66. (canceled)
70-72. (canceled)
74-75. (canceled)
78-83. (canceled)
85-87. (canceled)
Dependencies are inferred from the claim text.