Abstract: The subject matter disclosed herein is generally directed to methods and compositions for tagging cells of interest, tracking evolution of the tagged cells, and recovering the original tagged cells for further study. Specifically, cells are tagged with a DNA construct encoding a barcode sequence comprising a guide sequence. Barcoded cells can then be recovered using a reporter construct having CRISPR target sequences specific for the cell having a barcode of interest.

Abstract: Compounds are provided having the following structure (I) or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof, wherein G1, G1?, G2, G2?, G3, L1, L1?, L2, L2?, X, X?, Y and Y? are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.

Abstract: The invention relates to a composition comprising a double-stranded RNA (dsRNA) having two complementary strands, comprising at least one block of poly A and the complementary block of poly U, each strand having a length of between 50 and 200 bases, preferably between 55 and 200 bases, and a pharmaceutically acceptable vehicle, carrier or excipient, for use in a method of treating a cancer expressing a TLR3 receptor.

Abstract: Compounds are provided having the following structure (I) or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof, wherein X, Y, L1, L2, L3, G1, G2 and G3 are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.

Abstract: Provided herein are compositions and methods of reducing adduct formation.

Abstract: The invention provides an artificial sgRNA and a CRISPR/Cas9 system by combining the artificial sgRNA and Cas9. Activity of the sgRNA can be retained even when a nucleotide linker region for forming a single strand by linking the 3?-terminal of crRNA and the 5?-terminal of tracrRNA in sgRNA is substituted with an amino acid derivative linker, when the linker region existing between stem-loop 1 and stem-loop 2 of tracrRNA and/or the loop portion of stem-loop 2 are/is substituted with an amino acid derivative linker, or when an amino acid derivative linker is added/inserted into the vicinity of the 5?-terminal and/or the 3?-terminal of sgRNA. Stability in vivo can be improved by introducing one or more amino acid derivative linkers into the sgRNA.

Abstract: The present invention provides compositions and methods for treating cancer with peptide nucleic acid agents. In some embodiments, the present invention provides methods and compositions relating to peptide nucleic acid agents that target oncogenes. For example, the present invention provides compositions, including pharmaceutical compositions, comprising agents specific for BRAF V600E inhibition, or fragments or characteristic portions thereof. The present invention further provides various therapeutic and/or diagnostic methods of using BRAF V600E specific peptide nucleic acid agents and/or compositions.

Abstract: According to an aspect, provided are: a guide RNA; a vector comprising the same; a composition for removing a nucleic acid sequence encoding a KRAS polypeptide in the genome of a cell, containing the same; a composition for preventing or treating cancer, containing the same; and a method using the same. The present invention enables the mutation of a nucleic acid sequence encoding a KRAS polypeptide in the genome of a cell or a subject and, particularly, can be usable, as personalized or precision medical care, in the prevention or treatment of cancer.

Abstract: Method for preventing or treating neuropathic and/or inflammatory pain, wherein said method comprises administering at least one effective dose of the phosphorothioate oligonucleotide IMT504. The preventive method may be applied to a mammal to be subjected to a medical or surgical intervention, or to a mammal that may be injured performing a risky task (for example, a soldier in a battle) to prevent development of pain after a medical intervention or injury.

Abstract: The present disclosure relates to methods of assessing a sample of guide RNAs (gRNAs).

Abstract: Half of the cancers typically occur due to ineffective capability in their p53 proteins. The invention supplies the cells of the human body or patient with mRNA which will generate effective wild-type p53 proteins, so that this said protein can conduct proper surveillance inside the body's cells and act to destroy cells which have become or are about to become cancerous. The invention also includes introduction of interfering RNA to degrade mutated p53, allowing wild-type p53 proteins to form functional oligomers for cellular surveillance.

Abstract: The present invention discloses a method for modifying an amino acid attenuator, a class of amino acid attenuator mutants, engineered bacteria created on the basis of the amino acid attenuator mutants, and use of the engineered bacteria. The present invention protects a method for relieving the attenuation regulation of an amino acid operon gene, which is modification of the amino acid operon gene by: removing a gene coding for a leader peptide and an anterior reverse complementary palindromic sequence in the terminator stem-loop structure, and maintaining a posterior reverse complementary palindromic sequence in the terminator. The amino acid operon particularly can be histidine operon, tryptophan operon, phenylalanine operon, alanine operon, threonine operon and etc. The present invention can be used for the production of amino acids and derivatives thereof in fermentation by bacteria, providing a novel method for improving the production of amino acids in fermentation.

Abstract: The present disclosure provides RNA polymerase variants for high efficiency transcription.

Abstract: The present invention relates to compositions and methods of inhibiting p38 kinase to reduce gene and protein expression of DUX4 and downstream genes regulated by DUX4. The present invention further relates to methods for treating patients suffering from diseases associated with increased expression of DUX4 or expression of an aberrant form of DUX4, such as Facioscapulohumeral muscular dystrophy (FSHD).

Abstract: The present invention provides a compound represented by formula (I) usable as a cationic lipid that facilitates introduction of a nucleic acid, for example, into a cell, and a composition or the like containing the compound.

Abstract: A recombinantly expressed nucleotide triphosphate transporter efficiently imports the triphosphates of unnatural nucleotides into cells, and the endogenous cellular machinery incorporates those nucleotides into cellular nucleic acids. UBPs can therefore form within the cell's nucleic acids. Moreover, neither the presence of the unnatural triphosphates nor the replication of the UBP represents a significant growth burden. The UBP is not efficiently excised by nucleic acid repair pathways, and therefore can be retained as long as the unnatural triphosphates are available in the growth medium. Thus, the resulting cell is the first organism to stably propagate an expanded genetic alphabet.

Abstract: Disclosed are methods for performing transcription-dependent directed evolution (TRADE) and novel AAV capsids selected using such methods.

Abstract: De novo synthesized large libraries of nucleic acids are provided herein with low error rates. Further, devices for the manufacturing of high-quality building blocks, such as oligonucleotides, are described herein. Longer nucleic acids can be synthesized in parallel using microfluidic assemblies. Further, methods herein allow for the fast construction of large libraries of long, high-quality genes. Devices for the manufacturing of large libraries of long and high-quality nucleic acids are further described herein.

Abstract: The present invention relates to a use of leucine-zipper protein for diagnosis or treatment of fatty liver. According to the present invention, it could be confirmed that a particular fragment present at the leucine-zipper protein, especially, the N-terminal region thereof, plays an important role in the lipid metabolism in liver tissue by regulating transcriptional activity of Apolipoprotein A4, and therefore, the protein of the present invention or a fragment thereof can be utilized as a target for diagnosis, prevention, or treatment of fatty liver.

Abstract: An embodiment of the present disclosure can include a nanocarrier for delivering a cargo to a cell comprising: a bundle domain and a binding domain, the binding domain configured to bind to the cargo. An embodiment of the present disclosure can include a method for delivering a cargo to a cell comprising: introducing an amino-acid based nanocarrier to the cell, the nanocarrier can comprise an alpha-helical protein bundle domain and a binding domain, the cargo can bound to the binding domain and the cargo can be therapeutic cargo.