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.

The invention provides methods of treating an allergic inflammatory condition characterized by inflammation of the squamous epithelium of a target tissue by administering to the subject a pharmaceutical composition comprising a kallikrein 5 (KLK5) inhibitor, and a pharmaceutically acceptable carrier.

The invention provides methods of treating an allergic inflammatory condition characterized by inflammation of the squamous epithelium of a target tissue by administering to the subject a pharmaceutical composition comprising a kallikrein 5 (KLK5) inhibitor, and a pharmaceutically acceptable carrier.

Disclosed herein are methods of inhibiting nuclear pore complex assembly and inducing nuclear pore complex disassembly. Methods to screen for agents that inhibit nuclear pore assembly or induce nuclear pore complex disassembly are also disclosed.

This disclosure relates to oligonucleotides, compositions and methods useful for reducing PCSK9 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of PCSK9 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of PCSK9 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat hypercholesterolemia, atherosclerosis, and/or one or more symptoms or complications thereof.

This disclosure relates to oligonucleotides, compositions and methods useful for reducing PCSK9 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of PCSK9 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of PCSK9 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat hypercholesterolemia, atherosclerosis, and/or one or more symptoms or complications thereof.

The present disclosure provides a pharmaceutical composition for treating Alzheimer’s disease, which at least includes an extracellular vesicle that is prepared by a method including: a first culturing step: performing an amplification culture of an adipose-derived stem cell in a first culture medium at a cell density of 6,000-15,000 cells/cm.sup.2 until an amplification amount of the adipose-derived stem cell is above 90% of that before the culture; a second culturing step: culturing the amplified adipose-derived stem cell in a second culture medium at a cell density of 10,000-100,000 cells/cm.sup.2 for 20-30 hours; and an extracellular vesicle separating step: collecting a culture solution and separating the extracellular vesicle from the culture solution by utilizing a tangential flow filtration (TFF) or ultrafiltration method.

Disclosed are engineered retrons and methods of use such as to modify the genome of a host (e.g., mammalian) cell by delivering the engineered retron or the encoded ncRNA in vitro or in vivo to the host (e.g., mammalian) cell.

The present invention provides methods of treating, preventing or delaying the onset of bone marrow failure in Fanconi Anemia patients.

The present invention provides methods of treating, preventing or delaying the onset of bone marrow failure in Fanconi Anemia patients.