RU-2026109084-A - A variant of AAV that can infect the inner ear

Inventors

• КАМИЯ, Кадзусаку
• ТАНАКА, Йосинори
• Эноки, Тацудзи
• МИУРА, Тосиюки
• НАРИКИ, Хироаки
• ОКАМОТО, Сатико

Assignees

• ДЗЮНТЕНДО ЭДЬЮКЕЙШНЕЛ ФАУНДЕЙШН
• ТАКАРА БАЙО ИНК.

Dates

Publication Date

20260507

Application Date

20240910

Priority Date

20230911

Claims (13)

1. 1. A nucleic acid encoding a mutant capsid protein of an adeno-associated virus (AAV), wherein the mutant comprises a peptide comprising an amino acid sequence selected from the group consisting of SEQ ID NO: 1-7, or a peptide comprising an amino acid sequence that differs from the amino acid sequence selected from the group consisting of SEQ ID NO: 1-7 by the presence of a substitution, deletion, insertion and/or addition of one or more amino acids.
2. 2. The nucleic acid of claim 1, wherein the AAV capsid protein is derived from AAV2.
3. 3. The nucleic acid of claim 2, wherein the peptide is located at a position between amino acid number 588 and amino acid number 589 in VP1 of AAV2.
4. 4. Recombinant DNA containing a nucleic acid according to any one of claims 1-3.
5. 5. A cell containing a nucleic acid according to any one of claims 1-3.
6. 6. An AAV particle comprising a mutant AAV capsid protein that comprises a peptide comprising an amino acid sequence selected from the group consisting of SEQ ID NO: 1-7, or a peptide comprising an amino acid sequence that differs from the amino acid sequence selected from the group consisting of SEQ ID NO: 1-7 by the presence of a substitution, deletion, insertion and/or addition of one or more amino acids.
7. 7. The AAV particle of claim 6, wherein the AAV capsid protein is derived from AAV2.
8. 8. The AAV particle of claim 7, wherein the peptide is located between amino acids 588 and 589 of VP1 of AAV2.
9. 9. A method for producing a gene-transduced cell, comprising the step of bringing into contact with the cell an AAV particle containing a mutant AAV capsid protein containing a peptide with an amino acid sequence selected from the group consisting of SEQ ID NO: 1-7, or a peptide with an amino acid sequence different from the amino acid sequence selected from the group consisting of SEQ ID NO: 1-7, by the presence of a substitution, deletion, insertion and/or addition of one or more amino acids.
10. 10. The method of claim 9, wherein the AAV capsid protein is derived from AAV2.
11. 11. The method of claim 10, wherein the peptide is located at a position between amino acids 588 and 589 in VP1 of AAV2.
12. 12. A method for producing an AAV particle comprising using the cell of claim 5 as a host.
13. 13. A pharmaceutical composition comprising an AAV particle according to any one of claims 6-8 and a heterologous polynucleotide contained within the AAV particle.