288 results about "Genetic therapy" patented technology

A federated distributed computational system enables secure oncological therapy optimization through robotic integration. The system establishes a distributed graph architecture with secure communication channels connecting computational nodes, implementing encryption protocols for cross-institutional data exchange. Each node contains processing capabilities for fluorescence-guided imaging, uncertainty quantification, and expert knowledge integration while maintaining hierarchical knowledge graphs of oncological biomarkers, interventions, and outcomes. The system coordinates domain-specific knowledge through token-space communication and implements an advanced robotic integration system for surgical interventions using spatiotemporal tumor mapping, multi-modal fluorescence imaging, surgical robot coordination, and space-time stabilized mesh management. Key capabilities include wavelength-specific multi-modal fluorescence detection, combined epistemic and aleatoric uncertainty estimation, tensor-based data integration with adaptive dimensionality control, and light cone search for adaptive treatment optimization—all while maintaining strict privacy controls.

The invention relates to guide polynucleotides and methods for targeting and editing a portion of the 5' UTR-encoding region of genes encoding VGSC alpha subunits (NaV) to abrogate or create an upstream open reading frame (uORF). The invention relates to guide polynucleotides and methods for targeting and editing a portion of the 5' splice acceptor site (SA) of an exon of a gene encoding a voltage-gated sodium channel (VGSC) alpha subunit (NaV). The invention also relates to use of the guide polynucleotides and methods for treating genetic disorders, in particular Dravet syndrome.

Disclosed herein are embodiments of a polymer-based implant and methods of making and using the same. The polymer-based implant comprises a polymer component and a therapeutic agent. In some embodiments, the polymer-based implant can be used to treat and / or prevent retinal diseases and / or retinopathies. The polymer-based implant exhibits physical properties that provide the ability to safely place the polymer-based implant in an ocular region without undesired diffusion and also to allow for controlled and timely release of the therapeutic agent to a desired region of the ocular region, such as the retina. In particular disclosed embodiments, the polymer-based implant can be used for safe and effective gene therapy.

According to various aspects of this disclosure, the present disclosure relates to methods for reducing kidney inflammation, reducing kidney fibrosis, reducing oxidative stress in the kidney, preventing or reducing the likelihood of chronic kidney disease (CKD), treating or preventing acute kidney injury (AKI) or treating a kidney disease in a subject in need thereof comprising administering, e.g., intramuscularly, to the subject a recombinant adeno-associated virus (rAAV) vector, wherein the rAAV vector comprises a vector genome comprising a nucleotide sequence encoding a Fibroblast growth factor 21 (FGF21) or a functional fragment thereof and an AAV capsid (e.g., AAV1 serotype), optionally, wherein the subject does not suffer from diabetes.

The present invention provides an adeno-associated virus (AAV) vector gene therapy for use in treating a monogenic form of nephrotic syndrome, wherein the AAV vector comprises a NS-associated transgene and minimal nephrin promoter NPHIS1 or podocin promoter NPHIS2.

This present disclosure provides adeno-associated viral vectors, recombinant adeno-associated virus (rAAV), and methods of their use in gene therapy for treating propionic acidemia (PA). Also provided are pharmaceutical compositions comprising a recombinant adeno-associated virus of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the treatment of PA caused by mutations in propionyl-CoA carboxylase α-subunit (PCCA) or mutations in propionyl-CoA carboxylase β-subunit (PCCB).

The invention relates to gene therapy of ocular conditions. Described herein are compositions and methods for delivering therapeutic products, such as therapeutic proteins (e.g., antibodies), therapeutic RNAs (e.g., shRNAs, siRNAs, and miRNAs), and therapeutic aptamers, to the retinal / vitreous humor of the eye of a human subject to treat ocular conditions, involving, for example, recombinant viral vectors, such as recombinant adeno-associated virus (rAAV) vectors.

Provided herein is a nucleic acid encoding a prion protein, wherein the prion protein (a) comprises an amino acid sequence comprising a protective sequence variant such as a G127V mutation and (b) lacks a fully functional 3' glycosylphosphatidylinositol (GPI)-anchor attachment sequence. Also provided are viral vectors and pharmaceutical compositions comprising the nucleic acids. Further provided are methods and uses of treating prion diseases or other neurodegenerative diseases that confer toxicity through cell surface- anchored prion proteins in a subject, comprising administering to the subject an effective amount of a pharmaceutical composition or viral vector described herein.

A composition including a gene therapy delivery system and an alpha-galactosidase (GLA) polynucleotide encoding a GLA polypeptide, wherein the GLA polypeptide has at least 95% sequence identity to one of SEQ ID NOS. 1-40, 44-45, 51-52, 57, 61-173 or 212. Also provided are compositions, formulations, fusion proteins, and methods for the treatment of Fabry disease.

The invention belongs to the field of pathologies affecting the central nervous system: in particular, severe cerebral pathologies, more particularly those restricted by the presence of the blood-brain barrier (BBB): gliomas, cerebral metastases, neurodegenerative diseases (e.g. Alzheimer's, Parkinson's or ALS), genetic diseases (Huntington's, myopathies, Leigh syndrome, Rett syndrome), but also to the field of cancers, musculoskeletal and immunological disorders, vascular diseases (thrombus) in numerous organs (e.g. liver, kidney or muscle) and in combination with numerous therapeutic approaches (e.g. chemotherapy, immunotherapy, targeted therapy or gene therapy). The invention relates to a microbubble comprising a fluorinated polymer or copolymer and a fluorinated gas, to the use thereof and also to the polymer or copolymer intermediate compounds.

Provided herein are polynucleotides comprising a coding sequence for expression of a fibroblast growth factor 21 (FGF21) pathway activating agent. Also provided are vectors, recombinant viral genomes, and recombinant virus compositions comprising said polynucleotides, as well as associated methods.

Variants of coagulation factor VIII (FVIII) and expression cassettes encoding the FVIII variants thereof are described. A variant FVIII includes a glycoepitope of the FVIII protein including an N2118Q mutation. The N2118Q mutation can be combined with other mutations including a BDD-FVIII, N6, V3, RH, furin-cleavage site deletion. X10, K12, and / or F309S mutation to form additional FVIII variants. The FVIII variants with the N2118Q mutation and expression cassettes thereof can result in reduced immunogenicity of the resulting protein. When combined with other FVIII mutations, higher gene expression, increased secretion, increased stability, and higher FVIII functional activity can be achieved by the expressed FVIII variants. The variant FVIII and expression cassettes described here can be useful in protein replacement therapy and / or gene therapy for the treatment of hemophilia A.

The present invention provides an agent for protecting and regenerating pancreatic beta cells in a mammal having diabetes, the agent containing a recombinant viral vector that expresses hepatocyte growth factor (HGF), wherein the agent is characterized by being administered in a dose of 1010-1012 virus particles (vp) / kg body weight, and by the virus vector including a nucleic acid that encodes HGF downstream of a promoter having transcription activity capable of yielding a therapeutically effective HGF level in the blood at said dose.

The present invention provides gene therapies for the treatment of Friedreich's ataxia. Specifically, the present invention provides a nucleic acid, cloning vector and transfer vector for the production of an adeno-associated virus (AAV) vector. The nucleic acid comprises (i) a nucleic acid sequence encoding frataxin, (ii) a phospho-glycerate-kinase (PGK) promoter, and (iii) a woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The present invention also provides a pharmaceutical composition which comprises the AAV vector or nucleic acid. Also, the AAV vector, nucleic acid or pharmaceutical composition can be used as a medicament, specifically as a medicament for the treatment of Friedreich's ataxia.

The invention relates to means and methods for gene therapy of lysosomal storage disorders (LSDs), preferably a LSD with skeletal involvement, based on an ex vivo gene therapy approach comprising transduction of autologous hematopoietic stem and progenitor cells (HSPCs) with viral vectors for expressing enzymes that are deficient in the disorders. The final formulation is a suspension of transduced cells in culture medium for the administration to patients affected by the LSDs, preferably preceded by a conditioning regimen.

The present disclosure describes improved vectors, such as adeno-associated virus (AAV) vectors, for expressing progranulin and its variants in transduced cells, and the use of such vectors to increase the amount of progranulin in subjects with progranulin deficiency, such as certain subjects with frontotemporal dementia (FTD) or frontotemporal lobar degeneration (FTLD).

The disclosure provides for recombinantly modified Zika-based vectors that can be used in gene therapy applications.

We provide gene therapy for the treatment of muscular dystrophy, including but not limited to Duchenne muscular dystrophy (DMD). [Solution] This disclosure provides a recombinant adeno-associated virus (rAAV) comprising a nucleic acid molecule that delivers a nucleic acid encoding a U7-based snRNA, which is a nucleic acid that induces exon skipping for use in the treatment of muscular dystrophy, including but not limited to DMD, resulting from any mutation suitable for skipping exon 44 of the DMD gene (DMD exon 44), including but not limited to mutations involved in or affecting DMD exon 44.

The present invention features PPT1 polypeptides and encoding nucleic acid constructs. Uses of the polypeptides and encoding nucleic acid constructs include producing a PPT1 polypeptide, increasing PPT1 activity in a subject; and treating a PPT1-related disorder, such as CLN1 disease, in the subject.

The present invention is characterized by PPT1 polypeptides and coding nucleic acid constructs. The use of the polypeptides and coding nucleic acid constructs includes producing PPT1 polypeptides, increasing PPT1 activity in subjects, and treating PPT1-related disorders such as CLN1 disease in subjects.

The invention relates to a hybrid nanocarrier system comprising engineered metal nanoparticles (MeNP), a flavonoid (Fl), a Raman-active molecule (RAM), poly(allylamine hydrochloride) (PAH), genetic material (GM), and poly(styrene sulfonate) (PSS), designed for use in fields such as gene therapy, cancer treatment, chemotherapy, treatment of genetic diseases, innovative vaccine technologies, the production of innovative antibiotics for antibacterial and antimicrobial therapies, drug delivery, the dietary supplement market, Raman imaging systems, biotechnological applications, and the agrochemical industry.

The present invention relates to: an AAV-based PDE6B viral vector for treating retinitis pigmentosa, the AAV-based PDE6B viral vector containing a tissue-specifically expressed PDE6A promoter; and a use of thereof, and provides a gene therapy for treating retinitis pigmentosa caused by PDE6B gene deficiency. The in vivo therapeutic efficacy of seven types of AAV5-PDE6B vectors was confirmed using an AAV by using a PDE6A promoter that is tissue-specifically expressed in photoreceptor rod cells that develop retinitis pigmentosa. An AAV5-PDE6A-450-PDE6B vector was selected as a candidate due to exhibiting strong tissue-specific expression in photoreceptor rod cells under even off-target conditions, unlike the gene expression characteristics of an AAV5-CMV-PDE6B vector, and was tested so as to be usable in the development of a gene therapeutic agent for treating PDE6B-deficient retinitis pigmentosa patients. Therefore, the present invention, related to AAV5-PDE6B for retinitis pigmentosa treatment and containing a tissue-specifically expressed PDE6A promoter, provides retinitis pigmentosa patients with an important treatment option having improved safety, and can be expected to have fundamental therapeutic effects compared to conventional treatments.

The present disclosure is directed to compositions comprising mitochondrial optogenetics-based gene therapies and their methods of use. In some embodiments, a composition described herein comprises an expression vector comprising a first nucleic acid sequence encoding a channelrhodopsin fusion protein and a second nucleic acid sequence encoding a luciferase protein. In some cases, the first nucleic acid sequence and the second nucleic acid sequence are operably linked to an expression control sequence. In some instances, the channelrhodopsin fusion protein comprises a channelrhodopsin protein linked to an inner mitochondrial membrane-mitochondrial localization signal (IMM-MLS). In some implementations, when the expression vector is expressed, the luciferase protein is localized to the cytosol. In some cases, the IMM-MLS comprises a leading sequence from a mitochondrial inner membrane protein selected from ABCB10, ABCB140, Cytochrome C, and renal outer medullary potassium channel (ROMK).

A viral vector, wherein the viral vector comprises a COL4A3, a COL4A4, or a COL4A5 transgene.

The present disclosure relates to a novel ionizable lipid containing a biodegradable disulfide bond. The ionizable lipid containing a disulfide bond, according to the present disclosure, stably delivers an anionic drug when prepared into lipid nanoparticles and exhibits an excellent effect, in particular, in delivering nucleic acids, and thus can be effectively used in related technical fields such as lipid nanoparticle-mediated gene therapy.

This disclosure provides BDNF expression constructs and vectors comprising such constructs for treating metabolic disorders, including, but not limited to, obesity. The disclosed BDNF gene therapy is more effective than existing BDNF-based gene therapy constructs.

The disclosure relates to compositions and methods useful for gene therapy and for the prevention and treatment of cardiac allograft rejection in the transplant setting.

Compositions and methods for enhancing payload-based gene therapy by blocking binding of LDL to LDL receptor (LDLR) in the liver, and then administering a payload-based therapy targeting non-liver tissue to increase the percentage of payload of non-liver targets delivered to a subject.

Methods and compositions for gene therapy of retinal degeneration related to mutations in MER proto-oncogene, tyrosine kinase (MERTK).

The invention relates to gene therapy, and more specifically, to AAV gene therapy vectors containing a novel chimeric deoxyribonuclease (DNase) protein transgene and methods of treating ailments such as cancer and neurodegeneration.