Provided herein are aptamers capable of inhibiting the activity of Von Willebrand Factor (VWF). Pharmaceutical compositions comprising these aptamers are also provided. Methods of preventing blood clot formation in a subject by administering the aptamers are provided and methods of treating a blood clot by administering a VWF-targeting agent are also provided.

The present disclosure relates generally to cleavable linkers and uses thereof.

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 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.

Provided herein are aptamers capable of inhibiting the activity of Von Willebrand Factor (VWF). Pharmaceutical compositions comprising these aptamers are also provided. Methods of preventing blood clot formation in a subject by administering the aptamers are provided and methods of treating a blood clot by administering a VWF-targeting agent are also provided.

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 strand 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 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 strand 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.

Provided herein are photocleavable guide RNAs (gRNAs) comprising a targeting domain, a CRISPR RNA (crRNA) sequence for a CRISPR/Cas nuclease, and a at least one photocleavable moiety. Also provided herein are methods involving contacting a photocleavable gRNA and a CRISPR/Cas nuclease to form a CRISPR system. Also provided herein are methods involving providing a cell and contacting the photocleavable gRNA and a CRISPR/Cas nuclease, thus forming a ribonucleoprotein complex that binds to a target site sequence in the genome of the 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.

Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.