C12N2310/3511

COMPOSITIONS AND METHODS

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.

A CRISPR-CAS9 PLATFORM WITH AN INTRINSIC OFF-SWITCH AND ENHANCED SPECIFICITY
20220411790 · 2022-12-29 ·

A gene-editing system includes an engineered photocleavable guide RNA to endow Cas9 nuclease and base editing activities with a built-in mechanism for fast, light-mediated deactivation. In methods of use, the system retains high editing efficiency, natively improves specificity, offers precise spatial and temporal control, improves base editing purity through early deactivation.

LIGHT-INDUCIBLE CRISPR/CAS9 SYSTEM FOR GENOME EDITING
20220403378 · 2022-12-22 ·

A very fast and efficient CRISPR/Cas9 system is provided. Compositions include light-sensitive caged nucleotides at the PAM distal region of guide RNAs (gRNA.sup.caged) to create artificial mismatches as a “roadblock”. Upon light stimulation, the caging group (“roadblock”) is removed and the gRNA fully hybridizes with the target DNA. Thus, the pre-bound inactive Cas9/gRNA.sup.caged is rapidly converted to active Cas9.

Peptide nucleic acid conjugates
11597925 · 2023-03-07 · ·

The disclosure is directed to conjugates, e.g. PNA conjugates, as well as methods of employing the conjugates for detecting one or more targets in a biological sample, e.g. a tissue sample.

Particle-nucleic acid conjugates and therapeutic uses related thereto
09803197 · 2017-10-31 · ·

This disclosure relates to particles conjugated to therapeutic nucleic acids. In certain embodiments, the nucleic acid comprises a sequence that catalytically cleaves RNA, e.g., DNAzyme or RNAzyme. In certain embodiments, the particles contain nucleic acids with both DNAzyme and/or RNAzyme and siRNA sequences. The cleaving nucleic acids optionally comprise a sequence functioning to hybridize to a target of interest and/or the particles are further conjugated to a targeting moiety. In certain embodiments, conjugated particles are used in the treatment or prevention of cancer or viral infections or bacterial infections. In certain embodiments, conjugated particles are used in detecting metal ions and other small molecule analytes.

COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRESSION

Provided herein are oligomeric compounds with conjugate groups targeting apolipoprotein C-III (ApoCIII). In certain embodiments, the ApoCIII targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting ApoCIII for use in decreasing ApoCIII to treat, prevent, or ameliorate diseases, disorders or conditions related to ApoCIII. Certain diseases, disorders or conditions related to ApoCIII include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.

COMPOSITIONS AND METHODS FOR MODULATING TTR EXPRESSION

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.

PEPTIDE NUCLEIC ACID CONJUGATES
20230151361 · 2023-05-18 ·

The disclosure is directed to conjugates, e.g. PNA conjugates, as well as methods of employing the conjugates for detecting one or more targets in a biological sample, e.g. a tissue sample.

COMPOSITIONS AND METHODS FOR MODULATING TTR EXPRESSION

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.

Conjugated antisense compounds and their use

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.