The present disclosure provides components, compositions, methods, and systems thereof for nucleic acid editing. Particularly, the invention relates to adenine deaminases, fusion proteins of the adenine deaminases, systems including the adenine deaminases, and methods of using thereof.

The present invention relates to systems and methods for increasing expression of CD16 in immune cells to improve their proliferation and cytolytic functions and method of use to treat or prevent diseases or disorders.

The present invention provides a method for promoting sexual maturation of fish, the method including a step of suppressing functional expression of at least one of a leptin receptor and a leptin of the fish. An object of the present invention is to provide the method for promoting the sexual maturation of the fish in order to obtain individuals of fish capable of ovulation or spermiation at one year of age at a higher rate than in nature or under normal aquaculture conditions.

The present disclosure provides compositions and methods for prime editing with improved editing efficiency and/or reduced indel formation by inhibiting the DNA mismatch repair path way while conducting prime editing of a target site. Accordingly, the present disclosure provides a method for editing a nucleic acid molecule by prime editing that involves contacting a nucleic acid molecule with a prime editor, a pegRNA, and an inhibitor of the DNA mismatch repair pathway, thereby installing one or more modifications to the nucleic acid molecule at a target site with increased editing efficiency and/or lower indel formation. The present disclosure further provides polynucleotides for editing a DNA target site by prime editing comprising a nucleic acid sequence encoding a napDNAbp, a polymerase, and an inhibitor of the DNA mismatch repair pathway, wherein the napDNAbp and polymerase is capable in the presence of a pegRNA of installing one or more modifications in the DNA target site with increased editing efficiency and/or lower indel formation. The disclosure further provides, vectors, cells, and kits comprising the compositions and polynucleotides of the disclosure. The present disclosure also provides compositions and methods for prime editing with improved editing efficiency and/or reduced indel formation with modified prime editor fusion proteins. The disclosure further provides, vectors, cells, and kits comprising the compositions and polynucleotides of the disclosure.

A gene editing system for treating Duchenne muscular dystrophy, and a method for treating the disease using the gene editing system are disclosed. The system and method have the effects of making it possible to package the gene editing system in a single vector by editing the dystrophin gene using a CRISPR/Cas12f1 or TaRGET system, as well as making it possible to produce the dystrophin protein having a normal function by preventing the production of a stop codon of exon 51 through the skipping of exon 51, and thus can be useful for treating Duchenne muscular dystrophy.

Compositions and methods for treating a blood disorder in a subject comprising delivering a nucleic acid molecule including a nucleotide sequence encoding two to six guide RNAs (gRNAs) into a hematopoietic stem cell (HSC), a hematopoietic progenitor cell (HPC), or a population of hematopoietic stem and progenitor cells (HSPCs) are described.

The base editor systems (MOBE) that are derived from the CRISPR/Cas9 protein that enable to simultaneously introduce CG to TA and AT to GC point mutations at distinct genomic loci in living cells, with high efficiency and precision. In the MOBE disclosed herein, a piece of RNA (the gRNA) of the CRISPR/Cas9 protein is fused to the deaminase enzymes via a coat protein-aptamer interaction. A reporter plasmid comprising the MOBE system that allows for enrichment of cells with co-occurring orthogonal edits and increased editing efficiency.

Described herein are novel systems for targeting, editing or manipulating DNA in a cell or cell free environment, using novel type V B-GEn.16 (SEQ ID NO: 1) and variants thereof, as well as methods and kits for manipulating DNA.

Provided herein are recombinant adeno-associated virus (rAAV) compositions and methods for use of the rAAV encoding CasX proteins and guide ribonucleic acid (gRNA) sequences useful for nucleic acid sequence editing, and including transgene components. The rAAV may be delivered to cells to target a gene of interest.

The present disclosure provides methods and compositions concerning the CRISPR/Cas9 systems and associated guide RNAs, which target and excise portions of particular exons of a huntingtin gene, thereby abrogating huntingtin protein expression. The disclosure further provides methods and compositions for treating Huntington's Disease.