In an embodiment, the present disclosure relates to a method of editing at least one gene in plant target cells. The method generally includes introducing genetic components of a gene editing system to a first region of the plant. The genetic components are then transported from the first region to the second region, which is different from the first region. The genetic components are processed in the cells in the second region to form the gene editing system such that the gene editing system edits the at least one gene in the cells. The gene edited cells give rise to gametes that produce gene edited seeds upon fertilization.

The present disclosure provides methods and compositions for therapeutic use, where the methods and compositions include Type V CRISPR systems with RNA guides contain ribonucleotide bases and at least one deoxyribonucleotide base. The Type V CRISPR systems are used to perform therapeutic genome editing in somatic cells, induced pluripotency stem cells (iPSCs) and germline or embryonic cells of animals for xenotransplantation of organs and tissues.

Methods and compositions for use in treating subjects with non-syndromic progressive hearing loss caused by mutations of the miR96 by disruption of the mutant allele, and methods of use thereof, as well as genetically modified animals and cells.

Provided herein are methods and transgenic systems termed Ifegenia (Inherited Female Elimination by Genetically Encoded Nucleases to Interrupt Alleles) comprising transgenic animal strains encoding Cas9 and/or gRNA that targets a female essential gene which are capable of passing down these genes as well as mutant female essential genes in wild populations in order to suppress the population of the animals, as well as methods and systems for making such animals. In some instances, the methods and systems provided herein result in both somatic and heritable germline mutations of the female essential gene resulting in daughter killing, and female essential gene mutant males reproductively viable to pass along the female essential gene mutation and related transgenes into subsequent generations. The methods and systems are adaptable to population control of insects, in particular mosquitoes such as Anopheles gambiae.

Provided are variant Cas12f4 polypeptides, fusion proteins containing variant Cas12f4 polypeptides, nucleic acids encoding variant Cas12f4 polypeptides and fusion proteins, compositions and methods related to those variant Cas12f4 polypeptides and fusion proteins, and modified host cells comprising the variant Cas12f4 polypeptides and/or encoding nucleic acids. The variant Cas12f4 polypeptides disclosed herein exhibit improved functionality, stability, and/or other desirable properties and may be used in combination with guide Cas12f4 RNAs that can be used in a variety of applications ex vivo, in vitro, and in vivo.

The present invention relates to the technical field of genetic transformation of insect eggs. Specifically, the present invention refers to an efficient genetic editing system to obtain recombinant or genetically modified insect eggs, by incorporating genetic material directly into oocytes of female insects, which will then generate a large number of eggs with the incorporated or recombinant genetic material.

Disclosed herein include systems, devices, and methods for determining a protospacer sequence. For each of protospacer sequences, homology strings of the protospacer sequence can be generated. Each of the homology strings can be mapped to a reference sequence sequence to determine a match of the homology string in the reference sequence. Matches of one or more of the homology strings of can be filtered based on a protospacer adjacent motif (PAM) space to determine one or more off-target sites of the protospacer sequence. A profile of each protospacer sequence can be determined using the off-target sites of the protospacer sequence. A protospacer sequence can be selected based on its profile. A guide comprising the selected protospacer sequence can be designed and used for gene editing.

Systems, methods and compositions for targeting polynucleotides are detailed herein. In particular, engineered DNA-targeting systems comprising IsrB polypeptides, novel IsrB nucleases and reprogrammable targeting nucleic acid components and methods and application of use are provided.

Recombinant DNA molecules and constructs are provided that are useful for modulating gene expression in plants. One or more expression cassette(s) of a recombinant DNA molecule or construct may be excised from transgenic plants following transformation by the presence of flanking site-specific recombination sites in the recombinant DNA molecule or construct by expression of a recombinase enzyme encoded by the recombinant DNA molecule or construct. Such a recombinase system may be used to remove such expression cassette(s) from plants transformed with the recombinant DNA construct or vector. The recombinase transgene may be operably linked to a promoter for autoexcision in transformed plants without crossing to a different transgenic line expressing the recombinase. Methods for causing autoexcision of one or more expression cassette(s) in a transgenic plant, and plants and cells containing or transformed with a recombinant DNA molecule or construct of the present disclosure, are also provided.

Compositions for gene modification related to base editor systems, and methods of using the same to treat or prevent conditions associated with the extracellular deposition in various tissues of amyloid fibrils formed by the aggregation of misfolded transthyretin (TTR) proteins. Such conditions include, but are not limited to, polyneuropathy due to hereditary transthyretin amyloidosis (hATTR-PN) and hereditary cardiomyopathy due to transthyretin amyloidosis (hATTR-CM), both associated with autosomal dominant mutations of the TTR gene, and an age-related cardiomyopathy associated with wild-type TTR proteins (ATTRwt), also known as senile cardiac amyloidosis.