The present invention relates to the use of a DBait molecules by systemic routes without any combination with an endosomolytic agent.

Optimized inhibitory nucleic acids are provided. The nucleic acids have sequences which include an optimal inhibitory motif, such as GGG. Related methods are also described.

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as cerebral amyloid angiopathy (CAA) and early onset familial Alzheimer disease (EOFAD or eFAD), using such dsRNAi agents and compositions.

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the HMGB 1 gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a HMGB1 gene and to methods of N preventing and treating an HMGB1-associated disorder, e.g., metabolic disorder or non-alcholic fatty liver disease, e.g., non-alcoholic steatohepatitis (NASH).

The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second grand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended.

The subject invention provides methods, assays and products for detecting small-molecules in a sample, in particular, in both clinical and field settings. The method for detecting a small-molecule target in a sample comprises providing a sample, contacting the sample with an aptamer-based sensor selective for the small-molecule target, and sensitively and rapidly detecting the small-molecule target in the sample. Specifically, the method utilizes EATR-amplified small-molecule sensors based on cooperative binding split aptamers (CBSAs).

The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second grand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended.

Described herein are compositions that inhibit/degrade STAT3, and methods of using such compositions for chemoprevention of non-small cell lung cancer (NSCLC).

The invention provides aptamer-gene modulator conjugates, where the aptamer and the gene modulator are linked together. The invention further provides a method for cell-specific delivery of gene modulators to hard to transfect cells such as CD4+ cell.

Novel artificial exosomes and methods for producing novel artificial exosomes are provided. Methods of delivering cargo molecules to a cell using artificial exosomes are also provided.