This invention relates to compositions and methods for identifying the network that modulates, controls, or otherwise influences BCL-2 pathway inhibition, for example, energy-stress signaling, mitochondrial metabolism, vesicle transport, ribosomal components, and proteolysis. The invention also relates to identifying and modulating target genes and/or target gene products that modulate, control, or otherwise influence BCL-2 pathway inhibition.

Provided herein are compositions and methods of using base editors comprising a polynucleotide programmable nucleotide binding domain and a nucleobase editing domain in conjunction with a guide polynucleotide. Also provided herein are base editor systems for editing nucleobases of target nucleotide sequences.

The invention relates to compositions and methods for the preparation, manufacture, and therapeutic use of compositions comprising mRNA and a lipid nanoparticle comprising a compound of the invention and an ionizable lipid.

Disclosed herein are minimal arrestin domain containing protein 1 (ARRDC1) constructs, which drive the formation of ARRDC1-mediated microvesicles (ARMMs). These vesicles can be harnessed to package and deliver a variety of molecular cargos such as small molecules, nucleic acids, and proteins. An example of such cargo is the genome editor Cas9.

The present invention provides a method for preventing or treating liver cancer in a subject comprising administrating at least one selected from the group consisting of an Ssu72 peptide, a polynucleotide encoding the Ssu72 peptide and an expression vector containing the polynucleotide to the subject.

The present invention provides, among other things, improved methods for purifying I2S protein produced recombinantly for enzyme replacement therapy. The present invention is, in part, based on the surprising discovery that recombinant I2S protein can be purified from unprocessed biological materials, such as, I2S-containing cell culture medium, using a process involving as few as four chromatography columns.

Disclosed are methods and compositions for determining immunodominant peptides of target enzymes used in enzyme replacement therapy for lysosomal storage disorders. More specifically disclosed are immunodominant peptides for N-acetylgalactosamine-6-sulfatase (GALNS). Also disclosed are methods of inducing oral tolerance towards a target enzyme through oral administration of immunodominant peptides prior to commencing enzyme replacement therapy. More specifically disclosed is a method of inducing oral tolerance for GALNS, by orally administering specific immunodominant peptides for GALNS; in subjects suffering from mucopolysaccharidosis type IVA prior to commencing enzyme replacement therapy using GALNS.

The present invention relates to a pharmaceutical composition comprising a STT compound as an active ingredient for the prevention or treatment of Parkinson's disease. STT showed neuroprotective effect and apoptosis recovery effect in the Parkinson's disease cell model, restored the reduced motility in the MPTP animal model, and was shown to significantly protect dopamine cells, so it can be used for the prevention and treatment of Parkinson's disease.

Provided herein, inter alia, are compositions of short chain fatty acid (SCFA)-producing microorganisms and methods of making and using the same to inhibit pathogenic bacterial populations in the gastrointestinal tracts of an animal and additionally promote improvement of one or more metrics in an animal, such as increased bodyweight gain, decreased feed conversion ratio (FCR), improved gut barrier integrity, reduced mortality, reduced pathogen infection, and reduced pathogen shedding in feces.

Cationic polymers having one or more anionic ligand end groups, including a new class of carboxylated branched poly(beta-amino ester)s that can self-assemble into nanoparticles for efficient intracellular delivery of different biomolecules, including a variety of proteins is disclosed.