Compositions and Methods for Treatment of Diseases Associated with Trinucleotide Repeats in Transcription Factor Four
AssigneeIntellia Therapeutics, Inc.
InventorsSean Michael Burns, Bradley Andrew Murray, Sarah Beth Hesse
Life of this patent
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Abstract
This application relates to compositions and methods for excising trinucleotide repeats (TNRs) contained within intron 3 of TCF4, such as is seen in subjects having Fuchs endothelial corneal dystrophy (FECD), PSC, and Schizophrenia. Compositions comprising guide sequences targeting the alpha 2 subunit of collagen VIII are also disclosed for treatment of mutations therein that may contribute to FECD.
Claims37
A composition comprising at least one guide RNA comprising a guide sequence that directs a nuclease to a target sequence selected from SEQ ID NOs:
- 1-1084.
A composition comprising at least one guide RNA comprising a guide sequence that is identical or at least 99%, 98%, 97%, 96%, 95%, 94%, 93%, 92%, 91%, or 90% identical to a sequence selected from SEQ ID NOs:
- 1089-1278.
(canceled)
A composition comprising two guide RNAs selected from the following guide RNA pairings:
- a. a first guide RNA that directs a nuclease to SEQ ID NO:
- 83, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 109;
- b. a first guide RNA that directs a nuclease to SEQ ID NO:
- 85, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 109;
- c. a first guide RNA that directs a nuclease to SEQ ID NO:
- 86, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 112;
- d. a first guide RNA that directs a nuclease to SEQ ID NO:
- 85, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 125;
- e. a first guide RNA that directs a nuclease to SEQ ID NO:
- 86, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 109;
- f. a first guide RNA that directs a nuclease to SEQ ID NO:
- 85, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 107;
- g. a first guide RNA that directs a nuclease to SEQ ID NO:
- 83, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 125;
- h. a first guide RNA that directs a nuclease to SEQ ID NO:
- 86, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 125;
- i. a first guide RNA that directs a nuclease to SEQ ID NO:
- 86, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 107;
- j. a first guide RNA that directs a nuclease to SEQ ID NO:
- 64, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 106;
- k. a first guide RNA that directs a nuclease to SEQ ID NO:
- 85, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 114;
- l. a first guide RNA that directs a nuclease to SEQ ID NO:
- 86, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 114;
- m. a first guide RNA that directs a nuclease to SEQ ID NO:
- 83, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 114;
- n. a first guide RNA that directs a nuclease to SEQ ID NO:
- 53, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 114;
- o. a first guide RNA that directs a nuclease to SEQ ID NO:
- 83, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 112;
- and
- p. a first guide RNA that directs a nuclease to SEQ ID NO:
- 74, and a second guide RNA that directs a nuclease to SEQ ID NO:
- 114.
8-22. (canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
35-39. (canceled)
(canceled)
44-49. (canceled)
(canceled)
56-66. (canceled)
68-73. (canceled)
75-79. (canceled)
Dependencies are inferred from the claim text.