6567results about "SsDNA viruses" patented technology

The invention provides a modified TnpB-omega RNA gene editing system and application of the modified TnpB-omega RNA gene editing system. In particular, the present invention provides a modified TnpB related [omega] RNA skeleton. The modified omega RNA skeleton provided by the invention has at least three stem-loop structure fragments, and has a shorter length and a compact structure than a natural omega RNA skeleton, so that the modified omega RNA skeleton and TnpB can be jointly carried in a single AAV vector, the administration efficiency is improved, and high-efficiency gene editing is realized.

The disclosure relates to compositions and methods for the preparation, use, and / or formulation of adeno-associated virus capsid protein variants.

The invention belongs to the technical field of nano antibodies, and particularly relates to a nano antibody specifically bound with AAV9 and application of the nano antibody. The nano antibody provided by the invention can be specifically combined with the AAV9 adeno-associated virus without being combined with other AAV serotypes such as AAV2, AAV5 and AAV8, so that high-sensitivity and high-specificity detection of the AAV9 adeno-associated virus is realized. In practical application, the nano-antibody coupled solid-phase carrier provided by the invention is adopted as an affinity filler, the AAV9 adeno-associated virus can be effectively captured and mildly eluted, the virus purification and recovery effects are effectively improved, and the nano-antibody coupled solid-phase carrier is suitable for high-purity recovery of the AAV9 adeno-associated virus.

The disclosure relates to compositions and methods for the preparation, use, and / or formulation of adeno-associated virus capsid protein variants.

The invention belongs to the technical field of biological medicines, and provides application of DRAM1 as a diagnosis and treatment target for resisting non-alcoholic fatty liver disease related hepatocellular carcinoma in preparation of a kit, a carrier and a medicine aiming at the problem of lack of effective treatment targets of NAFLD-related HCC. As a target spot for resisting the non-alcoholic fatty liver disease related hepatocellular carcinoma, the expression level of the DRAM1 in liver tissues of non-alcoholic fatty liver disease related hepatocellular carcinoma patients and mice is increased. According to the application disclosed by the invention, research is developed aiming at the effect of the DRAM1 in the generation and development of NAFLD-related HCC, the analysis of TCGA liver cancer queue data and a GSE164760 data set is taken as an entry point, the influence of the DRAM1 on the diagnosis and prognosis of the liver cancer is comprehensively analyzed, and verification is carried out through a mouse in-vivo experiment, so that a new thought and a new target spot are provided for clinical diagnosis and treatment of the liver cancer.

The invention relates to the technical field of biological medicines, in particular to an anti-alopecia hair-nourishing small molecule peptide component and a preparation method thereof. Various small molecule peptides such as hair follicle targeting activation peptides and hair structure repairing peptides are designed and synthesized from the molecular level. Efficient synthesis and stable delivery of the peptide are realized through innovative preparation processes such as combination of solid-phase synthesis and enzyme digestion, ultrasonic-assisted purification, low-temperature freeze-drying and microcapsule wrapping integration and the like. The small molecule peptide component can achieve a synergistic effect in multiple aspects of activating hair follicle stem cells, repairing a hair structure, improving microcirculation and the like, has remarkable anti-hair-loss and hair-nourishing effects, is safe and free of side effects, reduces the cost by 30%-50%, and has a wide application prospect.

The disclosure is directed in part to variant capsid polypeptides that can be used to deliver payloads.

The present disclosure provides compositions and methods useful in the treatment of trinucleotide repeat disorders, including Huntington's disease and Friedreich's ataxia. The present disclosure also provides gRNAs designed to target the HTT or FXN genes. Complexes comprising a base editor and any of the gRNAs disclosed herein are also provided by the present disclosure. The present disclosure further provides polynucleotides, vectors, cells, compositions, and kits. Methods of treating Huntington's disease and Friedreich's ataxia are also provided herein.

The invention belongs to the field of virus detection and diagnosis, and relates to an anti-AAV2 monoclonal antibody as well as a preparation method and application thereof. The invention provides an anti-AAV2 rabbit monoclonal antibody, and amino acid sequences of a heavy chain variable region and a light chain variable region of the anti-AAV2 rabbit monoclonal antibody. The anti-AAV2 rabbit monoclonal antibody provided by the invention can be specifically combined with the VLP of AAV2, and can be used for detecting the AAV virus antigen. The anti-AAV2 monoclonal antibody provided by the invention provides an effective detection tool for the detection of AAV2 in the research and development process of a gene therapy vector and the detection of AAV2 in basic scientific research.

The disclosure is directed in part to variant capsid polypeptides that can be used to deliver payloads.

The invention provides an application of an AC2 gene and the like in improving the expression quantity of GFP (Green Fluorescent Protein) and / or inhibiting degradation of an mRNA (Messenger Ribonucleic Acid) attenuated substrate taking the GFP as a reporter gene. Researches find that AC2 has an mRNA decay inhibition function, can inhibit degradation of a nonsense-mediated mRNA decay substrate taking GFP as a reporter gene and degradation of an endogenous nonsense-mediated mRNA decay substrate of arabidopsis thaliana, can improve the expression quantity of mRNA of AT1G0106020, RPS6, SMG7, AT5G35490, AT5G64430, AT1G36730 and the like, can reduce the expression quantity of AT1G72450 and AT2G400, has interaction with Dcp1 and Dcp2, and can be used for inhibiting the expression quantity of mRNA of AT1G0106020, RPS6, SMG7, AT5G35490, AT5G6430, AT1G36730 and the like. The growth ability of the yeast in an auxotroph culture medium can be improved, the expression level of Dcp1 can be reduced, and a research result lays a foundation for clarification of AC2 functions.

Disclosed herein are genetically engineered cells for AAV production. The genetically engineered cell comprises molecular systems for temporal control of expression of genes required for AAV production. Also disclosed herein are methods of using genetically engineered cells for AAV production.

Polynucleotides, vectors, systems of vectors or polynucleotides, cells, and methods for expressing AAV Rep and Cap proteins are provided. In certain aspects, these polynucleotides, cells, and methods may produce higher levels of one or both of Rep and Cap transcripts. In some aspects, these polynucleotides, cells, and methods may produce higher levels of one or both of Rep and Cap proteins. In various aspects, these polynucleotides, vectors, systems of vectors or polynucleotides, cells, and methods use one or more heterologous polyA sequences with one or both of the Rep and Cap coding sequences. In numerous aspects, these polynucleotides, vectors, systems of vectors or polynucleotides, cells, and methods may use one or more enhancers to increase expression of Rep proteins, where such enhancers can be transcriptional enhancers, translational enhancers, or combinations thereof. In certain aspects, these polynucleotides, vectors, systems of vectors or polynucleotides, cells, and methods may be used to produce recombinant AAV (rAAV).

Disclosed are methods of treating a subject with a neurological disease associated with a splicing defect caused by TDP-43 proteinopathies, comprising administering to said subject an agent to increase expression levels and / or stability of hnRNP L, thereby attenuating and / or repairing the splicing defect. The disclosure also relates to nucleic acids targeting heterogeneous nuclear ribonucleoprotein L (hnRNP L), and their use.

In some aspects, the disclosure relates to compositions and methods useful for inhibiting SOD1 expression in cells (e.g., cells of a subject). In some embodiments, the disclosure describes isolated nucleic acids engineered to express an inhibitory nucleic acid targeting endogenous SOD1 and an mRNA encoding a hardened SOD1 protein. In some embodiments, compositions and methods described by the disclosure are useful for treating Amyotrophic Lateral Sclerosis (ALS) in a subject.

The invention discloses a novel microecological preparation composition for preventing and treating feline plague and application of the novel microecological preparation composition, and belongs to the technical field of microecological preparations. The recombinant lactobacillus plantarum NC8-VP2 constructed by a genetic engineering means can efficiently express the feline plague VP2 protein, and finally, a protective antigen expressed by the recombinant lactobacillus plantarum efficiently stimulates an organism to generate a specific antibody, so that the feline plague is prevented, and the use of chemical drugs is reduced. Recombinant escherichia coli BL21-pET32a-MLT constructed by the research can secrete and express melittin (MLT), large-scale production of melittin is realized, melittin and recombinant lactobacillus plantarum NC8-VP2 are further taken as active ingredients, a novel microecological preparation composition for preventing and controlling feline plague is developed and prepared, the morbidity of feline plague can be reduced, clinical symptoms of sick animals can be improved, and the novel microecological preparation composition has a good application prospect. Therefore, the compound has a relatively good application prospect in the aspect of preventing and treating feline plague.

The present invention provides constructs comprising modified riboswitches to regulate expression of a transgene within a subject. Methods of treating a disease, specifically an eye disease, are also contemplated.

Disclosed are AAV viral-based vector compositions useful in delivering a variety of nucleic acid segments, including those encoding therapeutic polypeptides to selected mammalian host cells for use in therapeutic autoimmune modalities, including, for example, the in vivo induction of immunological tolerance via a liver-directed AAV-based gene therapeutic regimen for treating and / or ameliorating autoimmune disorders such as multiple sclerosis. Further disclosed are nucleic acid segments encoding therapeutic polypeptides that have been codon-optimized for expression in human cells.

Provided herein are engineered nucleic acids (e.g., expression vectors, including viral vectors, such as lentiviral vectors, adenoviral vectors, AV vectors, herpes viral vectors, and retroviral vectors) that encode OCT4; KLF4; SOX2; or any combination thereof that are useful, for example, in inducing cellular reprogramming, tissue repair, tissue regeneration, organ regeneration, reversing aging, or any combination thereof. Also provided herein are recombinant viruses (e.g., lentiviruses, alphaviruses, vaccinia viruses, adenoviruses, herpes viruses, retroviruses, or AAVs) comprising the engineered nucleic acids (e.g., engineered nucleic acids), engineered cells, compositions comprising the engineered nucleic acids, the recombinant viruses, engineered cells, engineered proteins, chemical agents that are capable of activating expression of OCT4; KLF4; SOX2; or any combination thereof, an engineered protein selected from the group consisting of OCT4; KLF4; SOX2; or any combination thereof, an antibody capable of activating expression of OCT4; KLF4; SOX2; or any combination thereof, and methods of treating a (e.g., ocular disease), preventing a disease (e.g., ocular disease), regulating (e.g., inducing or inducing and then stopping) cellular reprogramming, regulating tissue repair, regulating tissue regeneration, or any combination thereof).

The present disclosure provides Crispr / cas9-based repressors for silencing gene targets in vivo and methods of use.

The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to tau.

The present letter invention provides an engineered AAV capsid in which at least one protein on the capsid is modified to include an n-mer motif that promotes transduction of the capsid into the central nervous system (CNS) by interaction with a transferrin receptor. Further embodiments provide a carrier system comprising one or more carriers encoding an AAV capsid and a method of delivering a cargo to a CNS. The methods comprise administering an AAV capsid according to embodiments described herein in vivo or in vitro, and the AVV capsid comprises one or more carrier molecules.

Disclosed herein are composition and methods of modifying a target double-stranded DNA (dsDNA) molecule in a cell. Compositions and methods are useful for circularizing a linear DNA plasmid (e.g., an AAV vector) and integrating it into a target genome. Systems, compositions, and methods may comprise a CRISPR-associated (Cas) protein, an RNA-dependent DNA polymerase, an LSR, and / or one or more guide nucleic acids or uses thereof.

The disclosure relates to compositions and methods for the preparation, use, and / or formulation of adeno-associated virus capsid protein variants.

A printed circuit board according to an embodiment comprises: an insulating layer; a circuit pattern disposed on the upper surface of the insulating layer; a support layer which is disposed on the upper surface of the insulating layer to expose the upper surface of the circuit pattern and is in contact with the sides of the circuit pattern; and a protective layer disposed on the upper surfaces of the support layer and the circuit pattern, wherein the upper region of the insulating layer comprises a first region and a second region, and the protective layer comprises an open region exposing the upper surfaces of the support layer and the circuit pattern that are disposed in the first region, and the support layer comprises a first upper surface positioned at the highest level among the upper surfaces of the support layer and a second upper surface positioned at the lowest level among the upper surfaces of the support layer, the second upper surface being lower than the first upper surface, and the protective layer comprises a first portion which contacts the upper surface of the circuit pattern of the first region and a second portion which contacts the upper surface of the support layer of the first region, and the second portion of the protective layer contacts the second upper surface of the support layer and includes a first lower surface which is lower than the upper surface of the circuit pattern.

Methods and compositions for modulating repeat non-ATG protein (RAN protein) translation are provided. In some aspects, the disclosure provides methods of inhibiting RAN protein translation by contacting a cell with an effective amount of an inhibitor of eIF2 phosphorylation or an inhibitor of protein kinase R (PKR). In some embodiments, methods described by the disclosure are useful for treating diseases associated with RAN protein translation, such as certain neurodegenerative diseases.

The present invention relates to the field of biomedicine, and discloses a fusion-type adeno-associated virus and its application in ear diseases and ophthalmic diseases. The fusion-type adeno-associated virus comprises a fusion peptide segment or a variant thereof formed by fusing peptide segments of serotypes AAV1, 2, 6 and 7, which can efficiently transduce hair cells and / or supporting cells in the cochlea, promote the regeneration of inner hair cells, and help restore hearing. The adeno-associated virus can also efficiently infect the RPE layer and is used for the treatment of ophthalmic diseases. As a safe vector, the fusion-type adeno-associated virus has broad application prospects in the treatment of ear diseases or ophthalmic diseases.

The invention provides a method for realizing fixed-point activation of a plant genome by directionally inserting an enhancer, and particularly, by inserting a short enhancer (STE) in a specific region of a target gene promoter, a target gene can be efficiently activated, the expression level of the target gene can be enhanced by about 2000 times, and the enhancement multiple can be flexibly adjusted as required. The site-specific activation method for the plant genome has high efficiency, wide applicability and stable heredity, and has outstanding application value in plant research and breeding.

The disclosure relates to compositions and methods for the preparation, use, and / or formulation of adeno-associated virus capsid protein variants.