Compositions and methods for adeno-associated viral production
AssigneeAsimov Inc.
InventorsJeremy J. Gam, Alec A.K. Nielsen
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Abstract
Disclosed herein are genetically engineered cells for AAV production. The genetically engineered cells comprise molecular systems for temporal control of expression of genes required for AAV production. Also disclosed herein are methods of using genetically engineered cells for AAV production.
Claims54
An Adeno-Associated Virus (AAV) production system comprising an expression control component and an AAV production component, wherein:
- (a) the expression control component comprises a polynucleic acid molecule encoding for a recombinase;
- and
- (b) the AAV production component comprises one or more polynucleic acid molecules collectively encoding for:
- Rep52 or Rep40;
- Rep78 or Rep68;
- E2A;
- E4Orf6;
- VARNA;
- VP1;
- VP2;
- VP3;
- and AAP;
- at least one of which comprises a nucleic acid sequence of an excisable element, wherein the excisable element comprises, from 5′ to 3′:
- (i) a first intron splice donor;
- (ii) a first recombinase site;
- (iii) a first intron splice acceptor;
- (iv) an exon comprising a stop codon;
- (v) a second intron splice donor;
- (vi) a second recombinase site;
- (vii) a second intron splice acceptor;
- wherein the first recombinase site and the second recombinase site correspond to the recombinase of (a).
An engineered cell comprising an Adeno-Associated Virus (AAV) production system having an AAV production component comprising one or more polynucleic acid molecules collectively encoding for:
- Rep52 or Rep40;
- Rep78 or Rep68;
- E2A;
- E4Orf6;
- VARNA;
- VP1;
- VP2;
- VP3;
- and AAP;
- at least one of which comprises a nucleic acid sequence of an excisable element, wherein the excisable element comprises, from 5′ to 3′:
- (i) a first intron splice donor;
- (ii) a first recombinase site;
- (iii) a first intron splice acceptor;
- (iv) an exon comprising a stop codon;
- (v) a second intron splice donor;
- (vi) a second recombinase site;
- (vii) a second intron splice acceptor.
A polynucleic acid molecule comprising a nucleic acid sequence having at least 80% identity with the nucleic acid sequence of any one of SEQ ID NOs:
- 134-139.
Dependencies are inferred from the claim text.