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US-2022090047-A1 · Mar 24, 2022 · US
Polynucleotides encoding engineered meganucleases having specificity for recognition sequences in the dystrophin gene
US12006522B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-12006522-B2 |
| Application number | US-202318360312-A |
| Country | US |
| Kind code | B2 |
| Filing date | Jul 27, 2023 |
| Priority date | Nov 12, 2020 |
| Publication date | Jun 11, 2024 |
| Grant date | Jun 11, 2024 |
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- Title
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Abstract
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The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.
First claim
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The invention claimed is: 1. A polynucleotide comprising a nucleic acid sequence encoding an engineered meganuclease that binds and cleaves a nucleic acid at a site that comprises a recognition sequence consisting of the nucleic acid sequence of SEQ ID NO: 6 in a dystrophin gene, wherein said engineered meganuclease comprises the amino acid sequence of SEQ ID NO: 43. 2. The polynucleotide of claim 1 , wherein said polynucleotide comprises a promoter operably linked to said nucleic acid sequence encoding said engineered meganuclease. 3. The polynucleotide of claim 2 , wherein said promoter is a muscle cell-specific promoter. 4. The polynucleotide of claim 1 , wherein said polynucleotide is an mRNA. 5. A recombinant DNA construct comprising a polynucleotide comprising a nucleic acid sequence encoding an engineered meganuclease that binds and cleaves a nucleic acid at a site that comprises a recognition sequence consisting of the nucleic acid sequence of SEQ ID NO: 6 in a dystrophin gene, wherein said engineered meganuclease comprises the amino acid sequence of SEQ ID NO: 43. 6. The recombinant DNA construct of claim 5 , wherein said polynucleotide comprises a promoter operably linked to said nucleic acid sequence encoding said engineered meganuclease. 7. The recombinant DNA construct of claim 6 , wherein said promoter is a muscle cell-specific promoter. 8. A recombinant virus comprising a polynucleotide comprising a nucleic acid sequence encoding an engineered meganuclease that binds and cleaves a nucleic acid at a site that comprises a recognition sequence consisting of the nucleic acid sequence of SEQ ID NO: 6 in a dystrophin gene, wherein said engineered meganuclease comprises the amino acid sequence of SEQ ID NO: 43. 9. The recombinant virus of claim 8 , wherein said recombinant virus is a recombinant AAV. 10. The recombinant virus of claim 9 , wherein said recombinant AAV has a capsid protein comprising the amino acid sequence of SEQ ID NO: 182 or a capsid comprising the amino acid sequence of SEQ ID NO: 183. 11. The recombinant virus of claim 8 , wherein said polynucleotide comprises a promoter operably linked to said nucleic acid sequence encoding said engineered meganuclease. 12. The recombinant virus of claim 11 , wherein said promoter is a muscle cell-specific promoter. 13. The recombinant virus of claim 9 , wherein said polynucleotide comprises a promoter operably linked to said nucleic acid sequence encoding said engineered meganuclease. 14. The recombinant virus of claim 13 , wherein said promoter is a muscle cell-specific promoter. 15. The recombinant virus of claim 10 , wherein said polynucleotide comprises a promoter operably linked to said nucleic acid sequence encoding said engineered meganuclease. 16. The recombinant virus of claim 15 , wherein said promoter is a muscle cell-specific promoter.
Assignees
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Classifications
Drugs for disorders of the muscular or neuromuscular system · CPC title
cell type or tissue specific enhancer/promoter combination · CPC title
for mammalian · CPC title
viral genome or elements thereof as genetic vector · CPC title
Viral vectors · CPC title
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Frequently asked questions
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- What does patent US12006522B2 cover?
- The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineer…
- Who is the assignee on this patent?
- Prec Biosciences Inc
- What technology area does this patent fall under?
- Primary CPC classification C12N9/22. Mapped technology areas include Chemistry & Metallurgy.
- When was this patent published?
- Publication date Tue Jun 11 2024 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 3 related publications on this page (citations in our corpus or others sharing the same primary CPC).