The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the DNAJB 1-PRKAC A fusion gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of DNAJB 1-PRKAC A fusion.

CRISPR/Cas-related compositions and methods which provide for efficient gene editing of eukaryotic cells using modified gRNAs.

Disclosed herein are STMN2 oligonucleotides with one or more spacers. In various embodiments, STMN2 oligonucleotides with spacer(s) reduce STMN2 transcripts with cryptic exon and increase full length STMN2 transcripts, thereby imparting therapeutic efficacy against neurological diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), or Alzheimer's disease (AD).

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.

The invention relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the SCAP gene, as well as methods of inhibiting expression of a SCAP gene and methods of treating subjects having a SCAP-associated disorder, such as nonalcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH), using such dsRNAi agents and compositions.

Described herein are aptamers capable of binding to human complement component 3 (C3) protein; compositions comprising a C3 binding aptamer with a C3-Protein; and methods of making and using the same.

The present disclosure provides oligomeric compound comprising a modified oligonucleotide having a central region comprising one or more modifications. In certain embodiments, the present disclosure provides oligomeric compounds having an improved therapeutic index or an increased maximum tolerated dose.

Chemical syntheses of guide molecules are disclosed, along with compositions and methods relating thereto.

The present disclosure relates to methods of administering, subcutaneously, to a subject, multimeric oligonucleotides having monomeric subunits joined by covalent linkers. The multimeric oligonucleotides have a molecular weight and/or size configured to increase in vivo activity of one or more subunits within the multimeric oligonucleotide relative to in vivo activity of the same subunit when administered in monomeric form of at least about 45 kD and other characteristics, such that their clearance due to glomerular filtration is reduced. The present disclosure also relates to such multimeric oligonucleotides and methods of synthesizing such multimeric oligonucleotides.

Described herein are aptamers capable of binding to human complement component 3 (C3) protein; compositions comprising a C3 binding aptamer with a C3-Protein; and methods of making and using the same.