Compositions and methods for integration of viral vectors
AssigneePoseida Therapeutics, Inc.
InventorsOscar ALVAREZ, Blair B. MADISON
Life of this patent
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Abstract
The present disclosure relates to compositions and methods for treating phenylketonuria (PKU). In particular, the present disclosure relates to AAV donor polynucleotide vectors and LNP compositions comprising a targeting gRNA pair and a nucleic acid encoding Cas-CLOVER fusion protein, and methods of using the compositions for treating PKU.
Claims22
An adeno-associated virus (AAV) donor polynucleotide comprising in the 5′ to 3′ direction:
- a) a first AAV ITR sequence;
- b) a first targeting sequence homologous to a first region of intron 3 of the albumin gene comprising the sequence of SEQ ID NO:
- 13;
- c) a splice acceptor sequence;
- d) a P2A sequence;
- e) a sequence encoding a first codon optimized and modified PAH gene comprising the sequence of SEQ ID NO:
- 25;
- f) a DNA spacer sequence;
- g) an inverted poly A sequence;
- h) an inverted sequence encoding a second codon optimized and modified PAH gene comprising the sequence of SEQ ID NO:
- 26;
- i) an inverted P2A sequence;
- j) an inverted splice acceptor sequence;
- k) a second targeting sequence homologous to a second region of intron 3 of the albumin gene comprising the sequence of SEQ ID NO:
- 23;
- and
- l) a second AAV ITR sequence.
An adeno-associated virus (AAV) donor polynucleotide comprising in the 5′ to 3′ direction:
- a) a first AAV ITR sequence;
- b) a first targeting sequence homologous to a first region of intron 3 of the albumin gene comprising the sequence of SEQ ID NO:
- 13;
- c) a TTRe promoter sequence;
- d) a splice acceptor sequence;
- e) a P2A sequence;
- f) a sequence encoding a codon optimized and modified PAH gene comprising the sequence of SEQ ID NO:
- 25;
- g) a 3′UTR sequence;
- h) a polyadenylation (poly(A)) sequence;
- i) a DNA spacer sequence;
- j) a second targeting sequence homologous to a second region of intron 3 of the albumin gene comprising the sequence of SEQ ID NO:
- 23;
- and
- k) a second AAV ITR sequence.
An AAV viral vector comprising the AAV donor polynucleotide of any of the preceding claims.
Dependencies are inferred from the claim text.