C12N2310/152

Compositions for enhancing targeted gene editing and methods of use thereof

Compositions and methods for enhancing targeted gene editing and methods of use thereof are disclosed. In the most preferred embodiments, gene editing is carried out utilizing a gene editing composition such as triplex-forming oligonucleotides, CRISPR, zinc finger nucleases, TALENS, or others, in combination with a gene modification potentiating agent such as stem cell factor (SCF), a CHK1 or ATR inhibitor, or a combination thereof. A particular preferred gene editing composition is triplex-forming peptide nucleic acids (PNAs) substituted at the γ position for increased DNA binding affinity. Nanoparticle compositions for intracellular delivery of the gene editing composition are also provided and particular advantageous for use with in vivo applications.

GENOME EDITING USING EFFECTOR OLIGONUCLEOTIDES FOR THERAPEUTIC TREATMENT
20200407727 · 2020-12-31 ·

The invention provides compositions and methods of making and using effector oligonucleotides, including effector oligonucleotides with greater than one mismatch as compared to its target sequence. These effector oligonucleotides are useful for improving the efficiency of genomic editing as well as providing therapeutic benefits to individuals in need thereof.

Hybrid oligonucleotides and uses thereof
10758558 · 2020-09-01 · ·

Provided herein are hybrid oligonucleotides comprising a region that promotes cleavage of a nucleic acid and a region that protects a nucleic acid from exonuclease activity. Such hybrid oligonucleotides are useful for modulating the expression of genes. Related compositions and methods are also provided. In some embodiments, methods are provided for treating a disease, such as by administering a hybrid oligonucleotide.

Genome editing using effector oligonucleotides for therapeutic treatment
10669547 · 2020-06-02 ·

The invention provides compositions and methods of making and using effector oligonucleotides, including effector oligonucleotides with greater than one mismatch as compared to its target sequence. These effector oligonucleotides are useful for improving the efficiency of genomic editing as well as providing therapeutic benefits to individuals in need thereof.

COMPOSITIONS AND METHODS FOR ENHANCING HOMOLOGOUS RECOMBINATION

The present disclosure generally relates to compositions and methods for improving the efficiency of homologous recombination. In particular, the disclosure relates to reagents and the use of such reagents.

METHODS AND COMPOSITIONS FOR MODULATING GENE EXPRESSION

The present technology relates to compositions and methods for modulating expression of genes which include a target oligonucleotide sequence, such as repeats of a particular oligonucleotide sequence containing 3 to 10 nucleotides. In particular aspects, the present technology relates to agents having a formula A-L-B, wherein -L- is a linker; A- is a Brd4 binding moiety; and -B is a nucleic acid binding moiety, such as a polyamide or complementary oligonucleotide, that specifically binds to the target oligonucleotide sequence.

ISOLATED DOUBLE STRANDED DNA POLYNUCLEOTIDE

The present invention relates to an isolated double stranded DNA polynucleotide that forms triplex with sequence 5-GGUGGCAGCAAGAGAAAAAUGAGGAAGAAGCAAAAGCGGAAA-3 (SEQ ID NO: 1) of the long non-coding RNA ANRIL (Antisense Non-coding RNA in the INK4 Locus). It also relates to a vector comprising the double stranded DNA polynucleotide, and to a pharmaceutical composition comprising the double stranded DNA polynucleotide or the vector. The present invention relates as well to the isolated double stranded DNA polynucleotide for use in the treatment of myocardial infarction, aneurysms, stenosis, myocardial infarction, aneurysms, cancers, eye diseases or type 2 diabetes.

Compositions and methods for enhancing homologous recombination

The present disclosure generally relates to compositions and methods for improving the efficiency of homologous recombination. In particular, the disclosure relates to reagents and the use of such reagents.

GENOME EDITING USING EFFECTOR OLIGONUCLEOTIDES FOR THERAPEUTIC TREATMENT
20240182906 · 2024-06-06 · ·

The invention provides compositions and methods of making and using effector oligonucleotides, including effector oligonucleotides with greater than one mismatch as compared to its target sequence. These effector oligonucleotides are useful for improving the efficiency of genomic editing as well as providing therapeutic benefits to individuals in need thereof.

METHODS AND COMPOSITIONS FOR MODULATING GENE EXPRESSION
20180280541 · 2018-10-04 ·

The present technology relates to compositions and methods for modulating expression of genes which include a target oligonucleotide sequence, such as repeats of a particular oligonucleotide sequence containing 3 to 10 nucleotides. In particular aspects, the present technology relates to agents having a formula A-L-B, wherein -L- is a linker; A- is a Brd4 binding moiety; and B is a nucleic acid binding moiety, such as a polyamide or complementary oligonucleotide, that specifically binds to the target oligonucleotide sequence.