A method for producing a potato plant with suppressed browning may use CRISPR/Cas9 system. In a method of producing a genome-edited potato plant with suppressed browning, a potato protoplast is transfected with a ribonucleoprotein complex containing sgRNA which targets StPPO2 (Solanum tuberosum Polyphenol Oxidase 2) gene derived from potato. Since the method involves no insertion of a foreign gene and includes only tiny mutation that is hardly distinguishable from natural mutation, it is expected that, unlike genetically modified organism (GMO) crops which require a huge amount of cost and time for evaluating the safety and negative environmental impacts, a great deal of cost and time can be saved in the present invention.

The present invention relates to a method, preferably a homology independent targeted integration (HITI), of integrating an exogenous DNA sequence into a genome of a cell comprising contacting the cell with a donor nucleic acid comprising said exogenous DNA sequence, optionally one or more albumin exons, wherein said donor nucleic acid is flanked at 5 and 3 by inverted targeting sequences; a complementary strand oligonucleotide homologous to the targeting sequence and a nuclease that recognizes the targeting sequence, wherein said targeting sequence is located at the 3 end of the albumin gene in a region selected from intron 12, intron 13 and intron 14 of said albumin gene. The invention also relates to systems, vectors and pharmaceutical compositions comprising said donor nucleic acid and/or complementary strand oligonucleotide homologous to the targeting sequence and/or nuclease and to medical uses thereof.

The present invention provides a drug which can be used as a pharmaceutical product for promoting epicardial cell regeneration and treating cardiac injury. In the present invention, a p21-inhibiting substance is used as an agent for promoting epicardial cell regeneration.

The present invention relates to a composition of lentiviral particles comprising a mutated integrase and an RNA-guided nuclease, for targeted gene insertion.

Strategies, systems, compositions, and methods for efficient production of knock-in cellular clones without reporter genes. An essential gene is targeted using a knock-in cassette that comprises an exogenous coding sequence for a gene product of interest (or cargo sequence) in frame with and downstream (3) of an exogenous coding sequence or partial coding sequence of the essential gene. Undesired targeting events create a non-functional version of the essential gene, in essence a knock-out, which is rescued by correct integration of the knock-in cassette, which restores the essential gene coding region so that a functional gene product is produced and positions the cargo sequence in frame with and downstream of the essential gene coding sequence.

Strategies, systems, compositions, and methods for genetically modifying cells to include one or more loss-of-function modifications and/or to include one or more gain-of-function modifications, as well as modified cells (and compositions of such cells) that include one or more loss-of-function modifications and/or that include one or more gain-of-function modifications, are described. In certain aspects, such modified cells include at least one gain-of-function modification within a coding region of an essential gene.

The disclosure provides a method for modulating gene expression in a cell-specific manner in response to an intracellular or extracellular stimulus. The disclosure provides a platform entitled the PROTEGE platform, which comprises a DNA binding module and a transcription factor binding module, referred to herein as PGM. The PGM binds the promoter region of a target gene with sequence specificity through the DNA binding module and also binds a TF through the TF-binding module. When the TF is activated in response to an intracellular or extracellular stimulus, it binds the PGM and, due to its close proximity to the promoter of the gene, modulates expression of a target gene in response to the stimulus.

Methods and materials for stimulating expression of target genes in plants are provided herein.

Screening assays are used to identify mutant homogentisate solanesyl transferase (HST) enzymes which are at least partially resistant to HST-inhibiting herbicides. Nucleic acids encoding the mutant HST enzyme proteins are made available and useful in modifying plants and plant parts so that they can be made herbicide resistant. Crop plants which express the mutant HST enzymes can be grown in the field and herbicides used to controlling unwanted other vegetation. The homogentisate solanesyltransferase (HST) enzyme or an active fragment thereof comprises the amino acid sequence motif: F[V/M]TX[F/Y] (SEQ ID NO: 1), wherein X is any amino acid; and wherein one or more of the amino acid residues of the motif are mutated.

Provided herein, inter alia, are gene therapy compositions and methods that can efficiently and safely restore MeCP2 gene function in subjects affected by Rett Syndrome.