Compositions of genetically modified beta-like cells are encompassed. Also encompassed are methods of treatment of type 1 diabetes using these compositions or compositions that inhibit the function of the identified genes.

A gene editing system comprising: (a) a Type V CRISPR nuclease polypeptide or a first nucleic acid encoding the Type V CRISPR nuclease polypeptide; (b) a reverse transcriptase (RT) polypeptide or a second nucleic acid encoding the RT polypeptide; (c) a guide RNA (gRNA) or a third nucleic acid encoding the gRNA, wherein the gRNA comprises one or more binding sites recognizable by the Type V CRISPR nuclease (CRISPR nuclease binding sites) and a spacer sequence specific to a target sequence within a genomic site of interest, the target sequence being adjacent to a protospacer adjacent motif (PAM); and (d) a reverse transcription donor RNA (RT donor RNA) or a fourth nucleic acid encoding the RT donor RNA, wherein the RT donor RNA comprises a primer binding site (PBS) and a template sequence.

The present disclosure provides methods of treating subjects having hypertension, coronary heart disease, and/or atrial fibrillation or at risk of developing hypertension, coronary heart disease, and/or atrial fibrillation, methods of identifying subjects having an increased risk of developing hypertension, coronary heart disease, and/or atrial fibrillation, and methods of detecting Solute Carrier Family 9 Isoform A3 Regulatory Factor 2 (SLC9A3R2) variant nucleic acid molecules and variant polypeptides.

The present disclosure provides methods of treating a subject having decreased bone mineral density or at risk of developing decreased bone mineral density, and methods of identifying subjects having an increased risk of developing decreased bone mineral density.

The present disclosure provides methods of treating a subject having a metabolic disorder or is at risk of developing a metabolic disorder or preventing a subject from developing a metabolic disorder, and methods of identifying subjects having an increased risk of developing a metabolic disorder.

The present disclosure provides methods of treating a subject having asthma or at risk of developing asthma, and methods of identifying subjects having an increased risk of developing asthma.

The present disclosure provides a high-throughput screening system and method for identification of novel drugs and drug targets. The method enables large-scale analysis of interactions between allogeneic pairs of target cells and immune cells by using an immune-bridge protein, library of guide RNA, and/or 3D tumor model.

The invention relates to peptide linkers and fusion proteins comprising linkers designed for optimizing the activity of the proteins comprised therein, and methods for using the same. The invention further relates to newly designed Cas12a-based cytosine base editors.

A mutant granule-bound starch synthase 1 (GBSS1) polypeptide and a nucleic acid, and use thereof are provided. Compared to an amino acid sequence of a parent GBSS1, the mutant GBSS1 polypeptide has a mutation at an amino acid corresponding to amino acid 427 and/or amino acid 428 of an amino acid sequence shown in SEQ ID NO: 1. An amylose content (AC) in a plant changes after the plant undergoes GBSS1 mutation, which has very promising application prospects in the improvement of edible quality of rice.

The present application provides materials and methods for treating a patient with one or more conditions associated with DMPK whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of DMPK gene in a cell by genome editing.