Abstract: Provided herein are RNA molecules encoding viral replication proteins and antigenic proteins or fragments thereof. Also provided herein are compositions that include RNA molecules encoding viral replication proteins and antigenic proteins or fragments thereof, and lipids. RNA molecules and compositions including them are useful for inducing immune responses.

Abstract: Lipid formulations that encapsulate messenger RNA (mRNA) are provided herein. The mRNA can be used to express CFTR protein in vitro or in vivo. The lipid formulations can be administered via inhalation to treat cystic fibrosis.

Abstract: Nucleic acid immunization is achieved by delivering a nucleic acid (NA), e.g., a mRNA or a DNA, encapsulated within a lipid-NA nanoparticle. The NA encodes an immunogenic compound of interest. The lipid-NA nanoparticle is effective for in vivo delivery of NA to a vertebrate cell, including upon administration to a subject. The lipid-NA nanoparticle are incorporated in pharmaceutical compositions for immunizing subjects against various diseases.

Abstract: Peptides and Peptide-lipid conjugates are provided in which the peptide has the general Formula (I) wherein, A1 is selected from serine, threonine, O—C1-6 alkyl serine, and O—C1-6 alkyl threonine; A2 is selected from serine, threonine, O—C1-6 alkyl serine, and O—C1-6 alkyl threonine; A3 is selected from glutamic acid, glutamine, asparagine, and aspartic acid; A4 is proline; each A5 is independently selected from a natural or modified amino acid; The peptide-lipid conjugates can be used in lipid formulations for the delivery of nucleic acids.

Abstract: A lipid composition containing a nucleic acid, wherein the lipid composition comprises a peptide-lipid conjugate, is provided. The peptide of the peptide-lipid conjugates can be from 4 to 52 amino acids in length. Methods of using the lipid composition in the in vivo delivery of nucleic acids are further provided.

Abstract: Nucleotides encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein are provided herein. Also describe are mRNA constructs that can be used to express CFTR protein in vitro or in vivo. The mRNA constructs can be formulated in a lipid formulation and administered via inhalation to treat cystic fibrosis.

Abstract: This disclosure encompasses compounds and compositions useful in methods for medical therapy, in general, for inhibiting expression of PDGFRB in a subject. The compounds have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers comprising UNA monomers and nucleic acid monomers.

Abstract: Provided herein are compositions methods of using liposomally encapsulated therapeutic drugs, such as staurosporine. Further provided herein are methods of using the liposome compositions to treat a cancer.

Abstract: Provided herein are novel compositions and methods to inhibit Olig2 activity. The Olig2 inhibitors and methods of using the same are useful, inter alia, for treating cancer. In particular the Olig2 inhibitors may be used to treat glioblastoma. Further, provided are peptide compositions capable of inhibiting Olig 2.

Abstract: Provided herein are compositions methods of using liposomally encapsulated therapeutic drugs, such as staurosporine. Further provided herein are methods of using the liposome compositions to treat a cancer.

Abstract: Provided herein are novel compositions and methods to inhibit Olig2 activity. The Olig2 inhibitors and methods of using the same are useful, inter alia, for treating cancer. In particular the Olig2 inhibitors may be used to treat glioblastoma. Further, provided are peptide compositions capable of inhibiting Olig 2.

Abstract: Provided herein are novel methods of making liposomally encapsulated drugs using reverse pH gradients and optimizing internal buffer compositions. Further provided herein are liposome compositions including an active pharmaceutical ingredient and uses thereof to treat a variety of diseases (e.g. cancer, inflammatory, neurological and cardiovascular diseases).