DCAS13-mediated therapeutic RNA base editing for in vivo gene therapy
AssigneeUniversity of Massachusetts
InventorsDan Wang, Guangping Gao, Jiaming Wang
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Abstract
Aspects of the disclosure relate to compositions (e.g., isolated nucleic acids, rAAV vectors, rAAVs, etc.) and methods for gene editing. The disclosure is based, in part, on isolated nucleic acids encoding combinations of gene editing proteins (e.g., Cas proteins) and base editors (e.g., Adenosine Deaminase Acting on RNA deaminase domains) with certain regulatory sequences that are amenable to packaging in recombinant adeno-associated viruses (rAAVs). In some embodiments, compositions described by the disclosure are useful for treating certain diseases in a subject in need thereof.
Claims11
An isolated nucleic acid comprising an expression cassette encoding:
- (i) a first region comprising a catalytically inactive Cas13b protein;
- (ii) a second region comprising a first signal sequence;
- (iii) a third region comprising an Adenosine Deaminase Acting on RNA deaminase domain (ADARdd);
- and
- (iv) a fourth region comprising a second signal sequence,
- wherein the expression cassette is flanked by adeno-associated virus (AAV) inverted terminal repeats (ITRs), and the size of the construct is less than 5 kb;
- wherein the first signal sequence or the second signal sequence is a nuclear localization signal (NLS) comprising the nucleic acid sequence set forth in SEQ ID NO:
- 4.
Dependencies are inferred from the claim text.