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Last Updated: March 28, 2024

Claims for Patent: 9,616,090


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Summary for Patent: 9,616,090
Title:Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
Abstract: The present disclosure is in the field of genome engineering, particularly targeted integration of a functional SCID-related genes (e.g., IL2RG, RAG1 and/or RAG2 gene) into the genome of a cell for provision of proteins lacking or deficient in SCID.
Inventor(s): Conway; Anthony (Richmond, CA), Cost; Gregory J. (Richmond, CA), Holmes; Michael C. (Richmond, CA), Urnov; Fyodor (Richmond, CA)
Assignee: Sangamo BioSciences, Inc. (Richmond, CA)
Application Number:14/813,924
Patent Claims:1. A nuclease comprising a DNA-binding domain and cleavage domain, wherein the DNA-binding domain is a zinc finger protein (ZFP), a TAL-effector domain, or a single RNA (sgRNA) and further wherein the DNA-binding domain binds to a target site in intron 1 of an endogenous IL2RG gene or intron 1 or 2 of an endogenous RAG gene such that the nuclease cleaves the IL2RG or RAG gene.

2. The nuclease of claim 1, wherein the DNA-binding domain comprises a zinc finger protein that binds to a sequence comprising a target site as shown in any of SEQ ID NOs:47-58 or 81-83.

3. The nuclease of claim 2, wherein the zinc finger protein comprises recognition helix regions as shown in a single row of Table 1.

4. The nuclease of claim 1, wherein the sgRNA comprises a DNA-binding guide RNA as shown in Table 5.

5. The nuclease of claim 1, wherein the TALE-effector domain comprises hypervariable diresidues (RVDs) as shown in in a single row of Table 3.

6. A polynucleotide encoding a nuclease according to claim 1.

7. A host cell comprising an exogenous sequence selected from the group consisting of: a sequence encoding an IL2RG polypeptide integrated into intron 1 of an endogenous IL2RG gene; a sequence encoding a RAG polypeptide integrated into intron 1 or 2 of an endogenous RAG gene; and combinations thereof, wherein the exogenous sequence is integrated into the host cell using a nuclease according to claim 1.

8. The host cell of claim 7, wherein the exogenous sequence comprises a cDNA selected from the group consisting of a sequence comprising exons 2 through 8 of a wild type IL2RG gene; a sequence comprising a full-length IL2RG gene; a sequence comprising exon 3 of a wild type RAG gene and a sequence comprising a full-length RAG gene.

9. The host cell of claim 7, wherein the cell is a hematopoietic stem cell or an induced pluripotent stem cell (iPSC).

10. A method for cleaving an endogenous SCID-related gene in a cell, the method comprising: introducing, into the cell, one or more polynucleotides according to claim 6 to the cell such that the nuclease(s) is(are) expressed and the one or more SCID-related genes are cleaved.

11. A method for targeted integration of a gene encoding a protein lacking or deficient in a subject with SCID, the method comprising: cleaving an endogenous SCID-related gene in a cell according to the method of claim 10 in the presence of a donor sequence encoding at least a functional fragment of the protein lacking or deficient in the subject with SCID such that the donor sequence is integrated into the endogenous SCID-related gene.

12. The method of claim 11, wherein the donor comprises a cDNA selected from the group consisting of a sequence comprising exons 2 through 8 of a wild type IL2RG gene; a sequence comprising a full-length IL2RG gene; a sequence comprising exon 3 of a wild type RAG gene and a sequence comprising a full-length RAG gene.

13. The method of claim 11, wherein the donor is carried on a viral or non-viral vector.

14. The method of claim 13, wherein the viral vector is an adeno-associated vector (AAV).

15. The method of claim 11, wherein the gene lacking or deficient in the subject with SCID is expressed under control of endogenous genetic control elements.

16. The method of claim 11, wherein the donor sequence comprises an exogenous promoter that drives expression of the gene lacking or deficient in the subject with a SCID.

17. A method of treating or preventing SCID in a subject, the method comprising administering a host cell according to claim 7 to the subject.

18. A kit comprising a polynucleotide according to claim 6.

19. A kit comprising a host cell according to claim 7.

Details for Patent 9,616,090

Applicant Tradename Biologic Ingredient Dosage Form BLA Approval Date Patent No. Expiredate
Merck Sharp & Dohme Corp. INTRON A interferon alfa-2b For Injection 103132 06/04/1986 ⤷  Try a Trial 2034-07-30
Merck Sharp & Dohme Corp. INTRON A interferon alfa-2b For Injection 103132 ⤷  Try a Trial 2034-07-30
Merck Sharp & Dohme Corp. INTRON A interferon alfa-2b Injection 103132 ⤷  Try a Trial 2034-07-30
>Applicant >Tradename >Biologic Ingredient >Dosage Form >BLA >Approval Date >Patent No. >Expiredate

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