Compositions and methods for enhancing donor oligonucleotide-based gene editing
Inventors
Quijano, Elias • Turchick, Audrey • Glazer, Peter
Assignees
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Publication Number
US-12441814-B2
Publication Date
2025-10-14
Expiration Date
Abstract
Compositions for enhanced gene editing and methods of use thereof are. The composition contains a cell-penetrating antibody and a donor oligonucleotide containing a sequence that can correct a mutation in a cell's genome. Preferably, the composition does not contain a nuclease, PNA, or nanoparticle. The compositions are used to modify the genome of a cell by contacting the cell with an effective amount of the composition. Genomic modification occurs at a higher frequency both ex vivo and in vivo, when cells are contacted with the cell-penetrating antibody and donor oligonucleotide as compared to the absence of the cell-penetrating antibody.
Core Innovation
The disclosed invention is a method of modifying the genome of a cell by contacting the cell with an effective amount of a composition that includes a non-covalent complex. The non-covalent complex comprises a donor oligonucleotide that can correct at least one mutation in the cell’s genome and an antibody or an antigen-binding fragment thereof.
The antibody or antigen-binding fragment contains specified VH and VL CDR amino-acid sequences, including VH CDR1, VH CDR2, VH CDR3, VL CDR1, VL CDR2, and VL CDR3. The composition excludes a nuclease, a triplex-forming peptide nucleic acid oligomer, and nanoparticles.
The approach is used for genome modification targeting mutations associated with particular genes and disease or disorder categories, and it is carried out in different contexts including in vivo. The dependent claim set further refines the target mutation categories, antibody format, donor oligonucleotide RNA form, and execution context, while maintaining the non-covalent complex and exclusion limitations.
Claims Coverage
The document provides one independent claim. The independent claim requires a genome-modifying method using a non-covalent complex of a donor oligonucleotide and an antibody or antigen-binding fragment with specified CDR sequences, while excluding a nuclease, a triplex-forming peptide nucleic acid oligomer, and nanoparticles.
Non-covalent complex of donor oligonucleotide and antibody or antigen-binding fragment
The method contacts a cell with an effective amount of a composition comprising a non-covalent complex including a donor oligonucleotide comprising a sequence that can correct at least one mutation in the cell’s genome and an antibody or an antigen-binding fragment thereof.
Specified antibody CDR sequences
The antibody or antigen-binding fragment comprises VH CDR1, VH CDR2, and VH CDR3 with amino-acid sequences of SEQ ID NO: 16, 17, and 18, and VL CDR1, VL CDR2, and VL CDR3 with amino-acid sequences of SEQ ID NO: 24, 25, and 26.
Exclusion of nuclease, triplex-forming peptide nucleic acid oligomer, and nanoparticles
The composition does not comprise a nuclease, a triplex-forming peptide nucleic acid oligomer, or nanoparticles.
Overall, the claim coverage centers on contacting cells with a non-covalent complex of a mutation-correcting donor oligonucleotide and an antibody or antigen-binding fragment defined by specific VH and VL CDR sequences, together with explicit exclusion of a nuclease, triplex-forming PNA oligomer, and nanoparticles.
Stated Advantages
Documented Applications
No documented applications found
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