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Systems, methods, and compositions for targeted nucleic acid editing

A polynucleotide, nucleotide sequence technology, applied in biochemical equipment and methods, pharmaceutical formulations, genetic material components, etc., can solve problems such as addressing

Pending Publication Date: 2020-07-14
THE BROAD INST INC +2
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0009] However, A→G and T→C point mutations represent only about 12% of known pathogenic SNPs, while G→A and C→T point mutations represent about 47% of known pathogenic SNPs, and cannot be decomposed by cytosine. Ammonia to address

Method used

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  • Systems, methods, and compositions for targeted nucleic acid editing
  • Systems, methods, and compositions for targeted nucleic acid editing
  • Systems, methods, and compositions for targeted nucleic acid editing

Examples

Experimental program
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preparation example Construction

[0571]Preparation of LNPs and CRISPR Cas encapsulation can be used and / or adapted from Rosin et al., Molecular Therapy, Vol. 19, No. 12, pp. 1286-2200, Dec. 2011). Cationic lipids 1,2-Dilinoleyl-3-dimethylammonium-propane (DLinDAP), 1,2-Dilinoleyloxy-3-N,N-dimethylaminopropane (DLinDMA), 1,2-Dilinoleyloxyketo-N,N-dimethyl-3-aminopropane (DLinK-DMA), 1,2-Dilinoleyl-4-(2-dimethylaminoethyl base)-[1,3]-dioxolane (DLinKC2-DMA), (3-o-[2″-(methoxypolyethylene glycol 2000)succinyl]-1,2-dimyristoyl -sn-ethylene glycol (PEG-S-DMG), and R-3-[(ω-methoxy-poly(ethylene glycol) 2000)carbamoyl]-1,2-dimyristoyloxy Propyl-3-amine (PEG-C-DOMG) can be provided or synthesized by Tekmira Pharmaceuticals (Vancouver, Canada). Cholesterol can be purchased from Sigma (St Louis, MO). Specific CRISPR CasRNA can be encapsulated in DLinDAP, DLinDMA, In the LNP of DLinK-DMA and DLinKC2-DMA (cationic lipid: DSPC: CHOL: PEGS-DMG or PEG-C-DOMG, the molar ratio is 40:10:40:10). When necessary, 0.2% SP-DiOC1...

Embodiment approach 1

[3048] Embodiment 1. A method of modifying an adenine in a target locus of interest, the method comprising delivering to the locus: (a) a Cpf1 nickase protein; (b) a guide molecule comprising a guide sequence for a direct repeat sequence; and (c) an adenosine deaminase protein or a catalytic domain thereof; wherein said adenosine deaminase protein or a catalytic domain thereof is covalently or non-covalently linked to said Cpf1 A nickase protein or said guide molecule, or adapted to be linked to said Cpf1 nickase protein or said guide molecule after delivery; wherein the guide molecule forms a complex with said Cpf1 nickase protein and directs said complex to bind The first DNA strand at the target locus of interest, wherein the guide sequence is capable of hybridizing to a target sequence comprising the adenine within the first DNA strand to form a heteroduplex, wherein the guide sequence is at the corresponding contains an unpaired cytosine at the position of the adenine, re...

Embodiment approach 2

[3049] Embodiment 2. The method of embodiment 1, wherein the adenosine deaminase protein or its catalytic domain is fused to the N- or C-terminus of the Cpf1 nickase protein.

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Abstract

The invention provides for systems, methods, and compositions for targeting and editing nucleic acids. In particular, the invention provides non-naturally occurring or engineered DNA-targeting systemscomprising a DNA-targeting Cpfl protein, at least one guide molecule, and at least one adenosine deaminase protein or catalytic domain thereof.

Description

[0001] Related Application and Incorporation by Reference [0002] This application claims U.S. Provisional Application No. 62 / 508,293, filed May 18, 2017, U.S. Provisional Application No. 62 / 561,663, filed September 21, 2017, U.S. Provisional Application No. 62 / 568,133, filed October 4, 2017 and priority to U.S. Provisional Application No. 62 / 609,957, filed December 22, 2017, each of which is incorporated herein by reference in its entirety. [0003] Statement Regarding Federally Funded Research [0004] This invention was made with government support under Grant Nos. MH100706 and MH110049 awarded by the National Institutes of Health. The government has certain rights in this invention. [0005] field of invention [0006] The present invention relates generally to systems, methods and compositions for targeting and editing nucleic acids, particularly for programmable deamination of adenines at target loci of interest. Background technique [0007] Recent advances in geno...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/10C12N9/22C12N9/78C12N15/62
CPCC12Y305/04004C12N9/22C12N9/78C12N15/102C12N15/62Y02A50/30A61K48/00C12N2310/533C12N15/11C12N15/79
Inventor F·张D·B·T·科克斯J·戈滕贝格O·O·阿布达耶B·蔡彻J·斯特雷克
Owner THE BROAD INST INC
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