18 results about "Genetic modulation" patented technology

Described herein are systems and methods for modulating gene expression. Also described herein are systems and methods for treating a disease or a condition by modulating gene expression.

Compositions and methods including stromal cells that have been genetically engineered to knock down or silence one or more genes are disclosed herein. The compositions include a population of isolated stromal cells that have been genetically engineered to reduce or eliminate expression one or more genes which confers permissivity to invasion (referred to herein as INVGOI) in fibroblasts, or cancer associated fibroblasts (CAFs). The cell compositions can be administered to a subject in need thereof to reduce or eliminate expression of one or more genes which confer permissivity to cancer invasion. Alternatively, inhibitory functional nucleic acids which specifically target one or more INV-GOI can be administered to a subject to treat cancer and in additional to chemotherapy, to sensitize cancer cells to chemotherapy.

The present disclosure relates to compositions including or obtained from genetically modified bacterial host cells (e.g., E. coli) and methods for using the same for cell-free bacteriophage synthesis (CFBS). In particular, the present technology relates to genetically modified E. coli that overexpress one or more of translation initiation factor IF-3 (infC), OxyS and CyaR and / or repress RecC subunit exonuclease RecBCD, and methods for using the same to obtain improved CFBS yields.

Described herein are systems and methods for modulating gene expression. Also described herein are systems and methods for treating a disease or a condition by modulating gene expression.

Provided herein are polypeptides, compositions comprising the polypeptides and methods for genome editing and gene regulation (e.g., activation and / or repression) using the polypeptides or the compositions comprising the polypeptides, such as, DNA binding domains derived from the genus of Legionella. These DNA binding proteins include a fragment of N-cap sequence and / or a C-cap sequence of a DNA binding protein derived from the genus of Legionella.

Described herein are systems and methods for modulating gene expression. Also described herein are systems and methods for treating a disease or a condition by modulating gene expression.

A novel gene modulation technique for modulating target nucleic acid and / or protein levels in cells, tissues, organisms and animals is disclosed. The new technology provides compositions for gene modulation applications, including prevention and treatment of human diseases. The composition comprises a short antisense RNA (asRNA) molecule having at least one deoxynucleotide monomer spacer motif. The invention further provides methods of using the compositions to modulate the expression or function of a target gene, to treat or prevent disease, and for biomedical research, disease diagnosis, and other biological applications.

The present disclosure provides engineered transcription factors for selectively upregulating SCN1a and their use in treating diseases and conditions such as Dravet syndrome. MicroRNA binding sites are also provided and their use for selective expression in parvalbumin neurons.

Described herein are systems and methods for modulating gene expression. Also described herein are systems and methods for treating a disease or a condition by modulating gene expression.

The invention relates to genomic mutations, and particularly, although not exclusively, to methods for detecting genomic mutations, which may be in coding or non-coding regions of a genome. In particular, the invention relates to methods for identifying multiple genomic or genetic mutations in conditions that may be associated with a complex genetic basis and / or epigenetic perturbations, and which provide a way to prioritise variants and reveal therapeutic insights and / or targets. The invention extends to methods of diagnosing disease characterised by genomic mutations, and also to methods of treating such conditions. The invention extends to computer-readable instructions, a computer-readable medium, and an apparatus for performing the methods described herein.

The invention provides a nucleic acid construct that is useful in directing RNA mediated gene regulation or RNA mediated gene editing. The invention further provides cells comprising the nucleic acid construct, and methods of using the same.

The present disclosure relates to compositions including or obtained from genetically modified bacterial host cells (e.g., E. colt) and methods for using the same for cell-free bacteriophage synthesis (CFBS). In particular, the present technology relates to genetically modified E. coli that overexpress one or more of translation initiation factor IF-3 (infC), OxyS and CyaR and / or repress RecC subunit exonuclease RecBCD, and methods for using the same to obtain improved CFBS yields.

Disclosed are abasic modification structures that can be incorporated into oligonucleotides to effectively modulate the expression of a target gene in a subject while mitigating off-target specificity. Also disclosed are gene modulators and pharmaceutical compositions comprising the abasic modification structures, as well as methods of using the structures to modulate the expression of a target gene while mitigating off-target specificity.

Provided herein are systems, kits, compositions, and methods for treating a subject with cancer or other disease with a CAR T-cell (or other immune cell) that has at least one endogenous gene that is modulated, and / or wherein mRNA from the gene is over-expressed or under-expressed, or that has exogenous mRNA that is expressed (e.g., expressed by an expression vector, or comprising modified bases). In certain embodiments, the endogenous gene, or mRNA therefrom, is inhibited or silenced and is selected from Table 2 (e.g., selected from PCNX1, PDCD10, MYC, ASXL1, RPTOR, BCAP31, ANKRD11, TCF3, IQCB1, CTLA4, ZZEF1, SRCAP, CARD8, DNMT1 and HSF2). In some embodiments, the endogenous gene, or mRNA therefrom, or mRNA in an expression vector, is over-expressed and is selected from Table 1 (e.g., NOSIP, FADD, RUNX1, AQR, ATE1, ATP9B, LSM2, CD3D, WNK1, EIF2B3, and TAL2).

Provided herein are DNA binding domains comprising a plurality of repeat units, wherein each repeat unit is expanded or contracted in length. Also provided herein are DNA binding domains comprising a plurality of repeat units, wherein each repeat unit is separated from a neighboring repeat unit by a linker. In certain aspects, the linker includes a recognition site. Also disclosed are DNA binding proteins that include a fragment of N-cap sequence of a TALE protein. The TALE protein may be a Xanthomonas TALE protein.

To provide pharmaceutical compositions for synergistically modulating microtubule associated protein tau (MAPT) gene expression in a cell.SOLUTION: A pharmaceutical composition comprises: a first genetic modulator which comprises a first DNA-binding domain that binds to a first target site of at least 12 nucleotides in the MAPT gene, and a first transcriptional regulatory domain or a first nuclease domain; and a second genetic modulator which comprises a second DNA-binding domain that binds to a second target site of at least 12 nucleotides in the MAPT gene, and a second transcriptional regulatory domain or a second nuclease domain; where the binding of the first genetic modulator to the first target site and the binding of the second genetic modulator to the second target site results in synergistic repression of MAPT gene expression.SELECTED DRAWING: Figure 13

To provide: clustered regularly interspaced short palindromic repeats (CRISPR) / CRISPR-based (Cas) 9-associated system related compositions; methods of using the CRISPR / Cas9-associated system related compositions for gene expression and genome engineering; compositions and methods of using the compositions for gene expression and genetic engineering in muscle, such as skeletal muscle and cardiac muscle.SOLUTION: A fusion protein comprises two heterologous polypeptide domains. The first polypeptide domain comprises a clustered regularly interspaced short palindromic repeats associated (Cas) protein. The second polypeptide domain has activity selected from the group consisting of transcription activation activity, transcription repression activity, transcription release factor activity, histone modification activity, nuclease activity, nucleic acid association activity, methylase activity, and demethylase activity.SELECTED DRAWING: Figure 1

Disclosed is an abasic modification structure which can be incorporated into an oligonucleotide to effectively modulate the expression of target genes in subjects while mitigating the non-target specificity. A gene modulating agent and a pharmaceutical composition comprising the abasic modification structure and a method of modulating the expression of a target gene while mitigating the non-target specificity by using the same are also disclosed.