A CRISPR/Cas system and method for editing or regulating transcription of a genome of a cell are provided, wherein the system includes a Cas endonuclease fused with one or more degron sequences and at least one activatable cognate single guide RNA harboring an inactivation sequence in a non-essential region of the cognate sgRNA, wherein said inactivation sequence comprises one or more endonuclease recognition sites of, e.g., a ribozyme.

Materials and methods for treating cancer (e.g., by reducing metastasis) are provided herein. For example, materials and methods for treating cancer by targeting WSB1 and/or pVHL are provided.

Methods of capturing N-glycan linked glycomolecules including N-glycans, N-glycopeptides and N-glycoproteins are described. The methods provide substantially unbiased capture of charged and uncharged N-glycans and/or N-glycan linked glycomoleules. Binding reagents for substantially unbiased binding of N-glycans and/or N-glycan linked glycomolecules are also described.

Provided herein and methods and kits for identifying inhibitors of SUMOylation. The provided methods include contacting a candidate agent with (i) a SUMO-conjugating peptide comprising an N-terminal linker and a detectable tag, and (ii) a SUMO comprising a metal complex, under conditions that allow binding between the SUMO-conjugating peptide and the SUMO, and detecting the detectable tag thereby determining the level of binding between the SUMO-conjugating peptide and SUMO. A reduced level of binding as compared to the level of binding in the absence of the candidate agent indicating the agent inhibits SUMOylation.

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of cosmetic polynucleotides, cosmetic primary transcripts and cosmetic mmRNA molecules.

Peptide vaccines against cancer are described herein. In particular, epitope peptides derived from the UBE2T that CTLs are provided. Isolated antigen-presenting cells with CTL inducibility and CTLs that target such peptides, as well as methods for inducing the antigen-presenting cell, or CTL are also provided. The present invention further provides pharmaceutical compositions containing such epitope peptides derived from UBE2T or polynucleotides encoding the polypeptides as active ingredients. Furthermore, the present invention provides methods for the treatment and/or prophylaxis of (i.e., preventing) cancers (tumors), and/or the prevention of a postoperative recurrence thereof, as well as methods for inducing CTLs, methods for inducing anti-tumor immunity, using the epitope peptides derived from UBE2T, polynucleotides encoding the peptides, or antigen-presenting cells presenting the peptides, or the pharmaceutical compositions of the present invention.

The disclosure provides nucleic acids (comprising AAV expression cassettes), AAV vectors, and compositions for use in methods for treating and/or delaying the onset of diseases associated with mutations in genes, such as UBE3A, associated with Angelman syndrome. Also, provided herein are methods for treating and/or delaying the onset of Angelman syndrome.

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.

The invention discloses peptides for the treatment and/or prophylaxis of diabetic retinopathy. The peptides of the invention comprise a glyceraldehyde-3-phosphate dehydrogenase (GAPDH) binding sequence and/or an E3 ubiquitin ligase seven in absentia homolog 1 (Siah1) binding sequence and an internalization sequence.

Peptide vaccines against cancer are described herein. In particular, epitope peptides derived from the UBE2T that elicit CTLs are provided. Isolated antigen-presenting cells with CTL inducibility and CTLs that target such peptides, as well as methods for inducing the antigen-presenting cell, or CTL are also provided. The present invention further provides pharmaceutical compositions containing such epitope peptides derived from UBE2T or polynucleotides encoding the polypeptides as active ingredients. Furthermore, the present invention provides methods for the treatment and/or prophylaxis of (i.e., preventing) cancers (tumors), and/or the prevention of a postoperative recurrence thereof, as well as methods for inducing CTLs, methods for inducing anti-tumor immunity, using the epitope peptides derived from UBE2T, polynucleotides encoding the peptides, or antigen-presenting cells presenting the peptides, or the pharmaceutical compositions of the present invention.