Compositions and methods disclosed herein can help provide improved delivery of non-natural therapeutic nucleotides for the treatment of diseases such as cancer. An example composition includes an assembly of amphiphilic polynucleotides, where each amphiphilic polynucleotide includes an aptamer portion, a first nucleotide portion, and a second nucleotide portion.

Compositions and methods are provided for the inhibition, treatment and/or prevention of degenerative myelopathy (DM) or amyotrophic lateral sclerosis (ALS).

Compounds and compositions for determining the level of antithrombin (ATIII) in a sample are described. Methods of forming the compounds and compositions are also described. Methods of using the compounds and compositions to quantify the level of ATIII in a subject are further described. The methods can be used to facilitate determining a dosage or heparin or ATIII to administer to a patient.

This disclosure provides compositions, recombinant expression constructs, and engineered systems that include a polynucleotide including or encoding a Cas12a tracrRNA, and methods for their use. The materials and methods of the disclosure are especially suited to sequence-specific genome editing of eukaryotic genomic sequences.

Some aspects of this disclosure provide compositions, methods, systems, and kits for controlling the activity and/or improving the specificity of RNA-programmable endonucleases, such as Cas9. For example, provided are guide RNAs (gRNAs) that are engineered to exist in an “on” or “off” state, which control the binding and hence cleavage activity of RNA-programmable endonucleases. Some aspects of this disclosure provide mRNA-sensing gRNAs that modulate the activity of RNA-programmable endonucleases based on the presence or absence of a target mRNA. Some aspects of this disclosure provide gRNAs that modulate the activity of an RNA-programmable endonuclease based on the presence or absence of an extended DNA (xDNA).

Object: To provide an antisense guide RNA for editing a target RNA by ADAR.

Solution: An antisense guide RNA for editing a target RNA by ADAR, containing at least one functional region and an antisense region that is complementary to a portion of the target RNA and can form a double strand with the target RNA, in which the at least one functional region is linked to the antisense region, and in which the guide RNA does not substantially contain an ADAR-recruiting base sequence.

The present invention provides an oligodeoxynucleotide which comprises a CpG oligodeoxynucleotide comprising a nucleotide sequence represented by formula (1):

5′X-CpG-L-CpG-TZ3′  (I) wherein X is T or C, L is a nucleotide sequence consisting of 1 to 7 bases, and Z is T or C, and consisting of 8 to 16 bases, and
a polydeoxyadenylic acid having a length capable of forming a complex with a β-1,3-glucan,
wherein the polydeoxyadenylic acid is linked to the 3′ side of the CpG oligodeoxynucleotide.

In addition, the present invention provides a complex containing said oligodeoxynucleotide and a β-1,3-glucan.

The disclosure provides for a targeting transfer RNA (ttRNA) that that suppresses nonsense mutations in messenger RNA, that comprises an anticodon sequence that binds to a stop codon and a variable loop sequence that comprises an RNA aptamer that has strong binding affinity to an RNA binding protein; and methods of use thereof.

Disclosed herein, in certain embodiments, are recombinant Arc and endogenous Gag polypeptides, and methods of using recombinant Arc and endogenous Gag polypeptides.

The present disclosure provides materials and methods for the delivery of therapeutic nucleic cells (and imaging agents) to tissues.