Abstract: The present disclosure relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The disclosure further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.

Abstract: The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids such as DNA. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.

Abstract: The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of DNA, RNA, and protein substrates. Each system includes one or more protein components and one or more nucleic acid components that together target DNA, RNA, or protein substrates.

Abstract: The present disclosure relates to cells (e.g., T cells) modified by Cas12i, methods of modifying the cells, processes for characterizing the modified cells, compositions and formulations comprising the modified cells, and uses of the compositions and formulations comprising the modified cells.

Abstract: The present disclosure relates to systems, compositions and methods for modifying target nucleic acid sequences.

Abstract: A method for inhibiting aberrant splicing in a Stathmin-2 (STMN2) transcript, the method comprising: genetically editing a STMN2 gene in a cell to delete (a) one or more nucleotides in a 3? splice site of intron 1, wherein the 3? splice site is adjacent to exon 2a, (b) one or more nucleotides in a region of intron 1 that is adjacent to the 3? splice site, or both (a) and (b), thereby inhibiting production of STMN2 transcripts including exon 2a and improving production of functional STMN2 transcripts in the cell. Also provided herein are gene editing systems for genetic modification of the STMN2 gene.

Abstract: The present disclosure relates to systems, compositions and methods for modifying target nucleic acid sequences.

Abstract: A system for genetic editing of a transthyretin (TTR) gene, comprising (i) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide, and (ii) an RNA guide or a second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an TTR gene. Also provided herein are methods for editing a TTR gene using the gene editing system disclosed herein and/or for treating diseases associated with the TTR gene.

Abstract: The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of DNA, RNA, and protein substrates. Each system includes one or more protein components and one or more nucleic acid components that together target DNA, RNA, or protein substrates.

Abstract: Provided herein are gene editing systems and/or compositions comprising RNA guides targeting HAO1 for use in genetic editing of the HAO1 gene. Also provide herein are methods of using the gene editing system for introducing edits to the HAO1 gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.

Abstract: The present invention relates to variant polypeptides, methods of preparing the variant polypeptides, processes for characterizing the variant polypeptides, compositions and cells comprising the variant polypeptides, and methods of using the variant polypeptides. The invention further relates to complexes comprising the variant polypeptides, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.

Abstract: 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.

Abstract: The present invention relates to compositions comprising RNA guides targeting TRAC, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.

Abstract: The present invention relates nucleases, processes for characterizing the nucleases, compositions comprising the nucleases, and methods of using the nucleases.

Abstract: The present invention relates to compositions comprising RNA guides targeting B2M, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.

Abstract: Provided herein are gene editing systems and/or compositions comprising RNA guides targeting HAO1 for use in genetic editing of the HAO1 gene. Also provide herein are methods of using the gene editing system for introducing edits to the HAO1 gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.

Abstract: The present disclosure relates to systems, compositions and methods for modifying target nucleic acid sequences.

Abstract: The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of a nucleic acid.

Abstract: The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of a nucleic acid.