Closed-end dna production with inverted terminal repeat sequences
AssigneeBioverativ Therapeutics Inc.
InventorsAjay Maghodia, Christian Mueller, Tongyao Liu
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 human bocavirus. Also disclosed are methods of using the nucleic acid molecules in gene therapy applications.
Claims37
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 SEQ ID NO:
- 1, and the second ITR comprises a polynucleotide sequence at least about 75% identical to SEQ ID NO:
- 2.
2-6. (canceled)
8-23. (canceled)
(canceled)
(canceled)
30-36. (canceled)
(canceled)
(canceled)
(canceled)
44-48. (canceled)
(canceled)
53-55. (canceled)
(canceled)
59-61. (canceled)
A method of treating a bleeding disorder comprising:
- administering a nucleic acid molecule to a subject in need thereof,
- wherein the nucleic acid molecule comprises the nucleotide sequence of SEQ ID NO:
- 3.
64-69. (canceled)
A recombinant bacmid comprising:
- a sequence encoding an HBoV1 Rep, wherein the inserted HBoV1 Rep sequence disrupts the reading frame of a reporter gene or functional portion thereof;
- and/or
- a heterologous sequence comprising a nucleic acid molecule comprising the nucleotide sequence of SEQ ID NO:
- 3, 9, 14, 33, or 35.
(canceled)
(canceled)
(canceled)
Dependencies are inferred from the claim text.