Compositions and methods are provided for disrupting biofilms formed by microbial organisms. In accordance with one embodiment such compositions are used in conjunction with standard treatment for use on chronic wounds. In one embodiment the biofilm disrupting composition comprises a nuclease and aurine tricarboxylic acid. The biofilm disrupting compositions disclosed herein can be used in conjunction with a therapeutic pharmaceutical composition comprising standard antibiotics.

The present invention includes compositions and methods for treating stroke in sickle cell anemia (SCA) patients. In certain embodiments, the patient is administered certain ENPP1- or ENNP3-containing polypeptides, mutants, or mutant fragments thereof.

The present invention relates to compositions and methods for targeting cancer-specific DNA sequences, such as copy number amplifications and other types of cancer-specific sequence variations, such as cancer-specific polymorphisms, insertions, or deletions. The present invention provides hereto sequence-specific DNA targeting agents targeting a sequence within the amplified DNA region or a sequence otherwise specific for a cancer cell compared to a non-cancer cell. The invention further relates to methods for treating cancer, comprising administering such sequence-specific DNA targeting agents. The invention further relates to methods for preparing sequence-specific DNA targeting agent, as well as screening methods using the DNA targeting agents.

A method of eliminating the risk of JCV activation in a subject undergoing immunosuppressive therapy, by administering an effective amount of a gene editing composition directed toward at least one target sequence in the JCV genome, cleaving the target sequence in the JCV genome, disrupting the JCV genome, eliminating the JCV infection, eliminating the risk of JCV activation, and treating the subject with an immunosuppressive therapy. A pharmaceutical composition including at least one isolated nucleic acid sequence encoding a CRISPR-associated endonuclease and at least one gRNA having a spacer sequence complementary to a target sequence in a JCV DNA, the isolated nucleic acid sequences being included in at least one expression vector. Pharmaceutical compositions including at least one isolated nucleic acid sequence encoding at least one TALEN, at least one ZFN, and gene editing composition of C2c1, C2c3, TevCas9, Archaea Cas9, CasY.1-CasY.6, CasX, or argonaute protein, which target at least one nucleotide sequence of the JCV genome.

Provided are amino acid sequences capable of binding to and inhibiting a Cas protein's ability to bind to a nucleic acid molecule, thereby inhibiting the Cas protein's function in genome editing. Such Cas protein inhibitors, which can be comprised of a major coat protein (G8P), an extracellular region of the G8P (G8P.sub.EX), or a biological equivalent, are useful in improving the specificity of Cas protein-based genome editing procedures.

The present invention features compositions and methods for editing deleterious mutations associated with alpha-1 anti-trypsin (A1AT) deficiency. In particular embodiments, the invention provides methods for correcting mutations in an A1AT polynucleotide using an adenosine deaminase base editor, ABE8, having unprecedented levels of efficiency.

The present invention relates to at least one delivery vehicle comprising: either a plurality of cleavaging agents that specifically recognize a plurality of nucleic acid sequences including unique sequences, which do not exist in normal cells, among variant region sequences specific for cells having genomic sequence variation in the cells having genomic sequence variation, or polynucleotides encoding the cleavaging agents; and a nuclease or a polynucleotide encoding the nuclease. The present invention also relates to a composition containing the delivery vehicle, and a method of inducing apoptosis of cells having genomic sequence variation by using the delivery vehicle or the composition.

The present invention relates to at least one delivery vehicle comprising: either a plurality of cleavaging agents that specifically recognize a plurality of nucleic acid sequences including unique sequences, which do not exist in normal cells, among variant region sequences specific for cells having genomic sequence variation in the cells having genomic sequence variation, or polynucleotides encoding the cleavaging agents; and a nuclease or a polynucleotide encoding the nuclease. The present invention also relates to a composition containing the delivery vehicle, and a method of inducing apoptosis of cells having genomic sequence variation by using the delivery vehicle or the composition.

The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

Provided are compositions related to HSD17B13 variants, including isolated nucleic acids and proteins related to variants of HSD17B13, and cells comprising those nucleic acids and proteins. Also provided are methods related to HSD17B13 variants. Such methods include methods for modifying a cell through use of any combination of nuclease agents, exogenous donor sequences, transcriptional activators, transcriptional repressors, and expression vectors for expressing a recombinant HSD17B13 gene or a nucleic acid encoding an HSD17B13 protein. Also provided are therapeutic and prophylactic methods for treating a subject having or at risk of developing chronic liver disease.