C12N2310/3531

Tuning CRISPR/Cas9 activity with chemically modified nucleotide substitutions

The present disclosure provides CRISPR/Cas9 ribonucleoprotein compositions comprising chemically modified CRISPR RNA (crRNA) guide and trans-acting CRISPR RNA (tracrRNA) components. Methods of using the disclosed CRISPR/Cas9 ribonucleoprotein compositions are also provided.

RNA interference compositions and methods for malignant tumors

This invention provides compositions for use in distributing active agents for treating a malignant tumor in a subject. The compositions contain RNAi molecules targeted to a human GST-π, along with RNAi molecules targeted to a human p21, and a pharmaceutically acceptable carrier. The carrier can include nanoparticles composed of an ionizable lipid, a structural lipid, one or more stabilizer lipids, and a lipid for reducing immunogenicity of the nanoparticles. This invention further provides methods for preventing or treating a malignant tumor by administering a therapeutically effective amount of an RNAi composition.

RNA interference compositions and methods for malignant tumors

This invention provides compositions for use in distributing active agents for treating a malignant tumor in a subject. The compositions contain RNAi molecules targeted to a human GST-π, along with RNAi molecules targeted to a human p21, and a pharmaceutically acceptable carrier. The carrier can include nanoparticles composed of an ionizable lipid, a structural lipid, one or more stabilizer lipids, and a lipid for reducing immunogenicity of the nanoparticles. This invention further provides methods for preventing or treating a malignant tumor by administering a therapeutically effective amount of an RNAi composition.

METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF ARGININOSUCCINATE LYASE (ASL)
20230295614 · 2023-09-21 ·

The present invention relates to methods and compositions for editing an ASL polynucleotide, e.g., an ASL polynucleotide comprising a SNP associated with argininosuccinate lyase deficiency. The invention also relates to methods and compositions for treating or preventing argininosuccinate lyase deficiency in a subject.

METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF ARGININOSUCCINATE LYASE (ASL)
20230295614 · 2023-09-21 ·

The present invention relates to methods and compositions for editing an ASL polynucleotide, e.g., an ASL polynucleotide comprising a SNP associated with argininosuccinate lyase deficiency. The invention also relates to methods and compositions for treating or preventing argininosuccinate lyase deficiency in a subject.

ACTIVATORS OF TYPE III CAS PROTEINS

Described herein are compositions and systems comprising activators of type III accessory nucleases and methods of using these compositions and systems.

ACTIVATORS OF TYPE III CAS PROTEINS

Described herein are compositions and systems comprising activators of type III accessory nucleases and methods of using these compositions and systems.

Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA
11447777 · 2022-09-20 · ·

This invention relates to compounds, compositions, and methods useful for reducing KRAS target RNA and protein levels via use of Dicer substrate siRNA (DsiRNA) agents possessing asymmetric end structures.

Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA
11447777 · 2022-09-20 · ·

This invention relates to compounds, compositions, and methods useful for reducing KRAS target RNA and protein levels via use of Dicer substrate siRNA (DsiRNA) agents possessing asymmetric end structures.

Methods of use of CRISPR CPF1 hybrid DNA/RNA polynucleotides

The present disclosure provides DNA-guided CRISPR systems; polynucleotides comprising DNA, RNA and mixtures thereof for use with CRISPR systems; and methods of use involving such polynucleotides and DNA-guided CRISPR systems.