Editing of VEGFR2 abrogated angiogenesis in two mouse models of oxygen-induced retinopathy (OIR) and laser-induced choroid neovascularization (CNV). Provided are compositions, e.g., Adeno-Associated Virus (AAV) Vectors comprising sequences encoding CRISPR/Cas9 proteins and guide RNA, and methods of use thereof for editing of Vascular endothelial growth factor receptor 2 (VEGFR2) gene to treat ocular disease associated with pathological angiogenesis, e.g., neovascular age-related macular degeneration (AMD), proliferative diabetic retinopathy (PDR) and retinopathy of prematurity (ROP).

A nutritional composition comprises (a) one to a plurality of physiologically acceptable calcium salts and/or chelates and (b) one or more phosphorus-containing components, such as phosphate salts, and/or phosphorus-rescuing components, such as phytase. The composition is useful for phosphorus-sparing calcium supplementation of the diet of a human subject, and for treatment of a low bone density condition in a human subject in need thereof.

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.

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 herein is a functional nucleic acid that includes i) one or more coding nucleotide sequences encoding a genome editing endonuclease; ii) regulatory sequences operably linked to the one or more coding nucleotide sequences; and iii) one or more genome editing endonuclease-recognized sequences, wherein the functional nucleic acid is configured to express the endonuclease in a host cell and thereby provide a cellular endonuclease activity in a sequence-specific manner in the host cell, and wherein cleavage of the functional nucleic acid by the endonuclease inactivates the cellular endonuclease activity. Methods of using the present functional nucleic acid, and systems and kits that find use in performing the same are also provided.

The present description relates to the use of a SRSF3 agent for regulating the function of a myeloid cell, such as a microglial cell and/or monocyte, for treating neurological conditions, cancers, bacterial infections and viral infections wherein the SRSF3 agent inhibits expression or function of SRSF3.

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.