1009 results about "Adeno-associated virus" patented technology

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.

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.

The invention relates to application of an MYZAP overexpression vector in preparation of a medicine for preventing and treating MIRI, and belongs to the technical field of biological medicine. In order to solve the problem that application of gene drugs in prevention and treatment of myocardial ischemia reperfusion injury is limited, the invention provides application of an MYZAP gene overexpression recombinant vector in preparation of drugs for prevention and treatment of myocardial ischemia reperfusion injury, and the MYZAP gene overexpression recombinant vector is an adeno-associated virus vector AAV9-MYZAP for overexpressing MYZAP. Experiments prove that MYZAP overexpression can improve the cardiac function after myocardial ischemia reperfusion, reduce the occurrence rate of arrhythmia and improve the functions of sodium ion channels, potassium ion channels and calcium ion channels of myocardial cells. On the basis, the invention further provides a medicine for preventing and treating myocardial ischemia reperfusion injury, a new strategy is provided for gene therapy of myocardial ischemia reperfusion injury, and the medicine has a wide clinical application prospect.

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

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

The invention relates to application of an MYZAP overexpression recombinant vector in preparation of a medicine for preventing and treating arrhythmia, and belongs to the technical field of biological medicines. In order to solve the problem that application of gene drugs in prevention and treatment of arrhythmia after myocardial infarction is limited, the invention provides application of a myocardial microbelt adhesion protein MYZAP overexpression recombinant vector in preparation of drugs for prevention and treatment of arrhythmia, and the MYZAP overexpression recombinant vector is an adeno-associated virus vector AAV9-MYZAP of an overexpression MYZAP gene. In-vivo experiments prove that MYZAP overexpression can reduce the occurrence of arrhythmia after myocardial infarction by increasing the content of MYZAP protein in cardiac myocardial cells, and improve cardiac functions and cardiac electrical conduction disorders after myocardial infarction. On the basis, the invention further provides a medicine for preventing and treating arrhythmia after myocardial infarction, a new strategy is provided for gene therapy of arrhythmia after myocardial infarction, and the medicine has a wide clinical application prospect.

The present invention relates to an exogenous target gene expression cassette for delivering an exogenous target gene to the retina, in particular AIPL1 to retinal pigment epithelial cells and photoreceptor cells, comprising an IRBP enhancer sequence, a rhodopsin kinase (RK) promoter sequence and a CAG intron sequence, which are operatively linked, and an exogenous target gene. The present invention also relates to a recombinant adeno-associated viral vector comprising a viral capsid comprising a capsid protein or a capsid protein variant and a viral vector genome comprising an expression cassette encoding for specifically expressing an exogenous target gene in retinal pigment epithelial cells and photoreceptor cells. The recombinant adeno-associated virus vector can be used for relieving or treating retinal degenerative eye diseases by intravitreal administration or subretinal administration.

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 AAV2 and application of the nano antibody. The nano antibody provided by the invention can be specifically combined with the AAV2 adeno-associated virus without being combined with other AAV serotypes such as AAV5, AAV8 and AAV9, so that high-sensitivity and high-specificity detection of the AAV2 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 AAV2 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 AAV2 adeno-associated virus.

The invention belongs to the technical field of antibody preparation, and particularly relates to a nano antibody for resisting different serotypes of adeno-associated viruses and application of the nano antibody. The nano antibody is selected from one of Nb1 to Nb24; the amino acid sequences of the heavy chain variable regions of the nano antibodies Nb1 to Nb24 are respectively shown as SEQ ID NO. 1 to 24. The nano antibody provided by the invention can sensitively and specifically bind to AAV, has excellent broad-spectrum recognition and binding capacity to various serotypes such as AAV2, AAV5, AAV8 and AAV9, and has a good application prospect in the fields of AAV immunodetection, affinity purification and the like.

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

The disclosure relates to compositions and methods for, inter alia, altering, e.g., enhancing, the level of GBA1 protein via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful, inter alia, for the treatment of subjects who have, have been diagnosed with having, or are at risk of having a GBA1-related disorder, e.g., Parkinson's Disease (PD), Gaucher Disease (GD), Parkinson's Disease Dementia (PDD), Dementia with Lewy Bodies (DLB), or Lewy Body Dementia (LBD).

The present disclosure relates to a truncated ATP7B in which CRE5 and / or CRE6 is present, optionally CRE1 and / or CRE2 is present. The present disclosure also provides an adeno-associated viral vector encoding the truncated ATP7B for use in gene therapy for the treatment of Wilson's disease (WD).

The disclosure relates to small interfering RNA (siRNA) molecules targeting MAPT and adeno-associated viral (AAV) particles encoding the same for treating tauopathies.

Adeno-associated virus (AAV) particles that are redirected with an antibody against CACNG1 and carry a nucleotide of therapeutic interest are provided. Also provided are methods of making and using the AAV particles, e.g., for treating a patient in need thereof or for manufacturing a medicament for treating a patient in need thereof.

An engineered producer cell comprising an inactivating mutation in one or more endogenous REP binding sites is provided, as are methods for producing the engineered producer cell and using the engineered producer cell to produce a recombinant viral vector and reduce producer cell genomic DNA contamination of a recombinant adeno-associated virus vector preparation.

The disclosure relates to compositions and methods for modulating, e.g., reducing or eliminating, the expression of ATXN2 via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, suspected of having, or having spinocerebellar ataxia type 2 or another ATXN2-related disorder.

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 HER2.

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

The presently described and claimed disclosure relates to capillary electrophoresis methods for quantifying an intact AAV genome and protein components in an AAV using the same capillary electrophoresis system. The claimed and described approach offers an automated analysis of AAV samples and provides information to determine the AAV empty / full ratio.

The invention belongs to the field of biotechnology and biomedicine, and discloses an AAV system suitable for PFIC3 gene therapy and a preparation method. The system is composed of two adeno-associated virus vectors. The packaging plasmid of the adeno-associated virus vector comprises an auxiliary packaging plasmid pDP8.ape containing an AAV2Rep gene and an AAV8Cap gene, and a vector plasmid pAB288-Alb-hABCB4 for expressing an ABCB4 gene of human MDR3 protein, a self-cleavage protein P2A, a mouse albumin gene homologous arm sequence and an ITR sequence of AAV2, the packaging plasmid of the other adeno-associated virus vector comprises an auxiliary packaging plasmid pDP8.ape containing an AAV2Rep gene and an AAV8Cap gene, and a vector plasmid pX602-saCas9-gRNA for expressing a saCas9 protein and a sgRNA of a targeted mouse albumin genome.

Provided herein are methods of expressing syntaxin-binding protein 1 (STXBP1) in a neuron. Also provided herein is a modified adeno-associated virus (AAV) encoding a STXBP1 under the control of a promoter operable in a brain cell or neuronal cell.

The disclosure relates to compositions, methods, and processes for the preparation, use, and / or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.

The disclosure relates to compositions and methods for altering, e.g., enhancing, the level of CDKL5 protein via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of a CDKL5-related disorder, e.g., a CDKL5-related neurodegenerative or neuromuscular disorder.

The invention relates to the technical field of medicines, and discloses six adeno-associated virus (AAV) injections carrying novel artificial chemical genetics receptor gene sequences, and a combinatorial chemical genetics scheme formed by the injections and a small molecule agonist quetiapine. Compared with the traditional chemical genetics, the medicine quetiapine with smaller clinical side effect is used as the activating agent for the first time, and a novel chemical genetics system with lower medication risk is developed. The system can effectively reduce the excitatory activity of neurons, avoids the medication risk caused by clozapine or other non-market traditional chemical genetics activators, and improves the feasibility of clinical transformation of the chemical genetics system. In the aspect of application of a novel or traditional chemical genetics system, the combined treatment scheme is applied to intervene in Parkinson's disease (PD) for the first time, abnormal activities of STN and related loops of PD patients can be inhibited, and the dyskinesia symptom can be remarkably improved.

The invention relates to the technical field of medicines, and discloses an adeno-associated virus (AAV) injection carrying an artificial chemical genetics receptor gene sequence, and a combinatorial chemical genetics scheme formed by the adeno-associated virus injection and a small molecule agonist quetiapine. Compared with the traditional chemical genetics, the medicine quetiapine with smaller clinical side effect is used as the activating agent for the first time, and a novel chemical genetics system with lower medication risk is developed. The system can effectively reduce the excitatory activity of neurons, avoids the medication risk caused by clozapine or other non-market traditional chemical genetics activators, and improves the feasibility of clinical transformation of the chemical genetics system. In the aspect of application of a novel or traditional chemical genetics system, the combined treatment scheme is applied to intervene in Parkinson's disease (PD) for the first time, abnormal activities of STN and related loops of PD patients can be inhibited, and the dyskinesia symptom can be remarkably improved.

Aspects of the disclosure relate to compositions and methods for base editing using viral vectors. In some aspects, the disclosure provides an adenine base editor (ABE) utilizing an evolved Cas9 nickase derived from Neisseria meningitidis; and adeno-associated viruses (AAVs) comprising (or encoding) such adenine base editors. In some aspects, compositions described herein are useful for treatment of certain diseases, for example pulmonary diseases.

The present invention provides novel gene therapy compositions for use in treatment of Duchenne Muscular Dystrophy (DMD).

The disclosure relates to compositions and methods for, inter alia, altering, e.g., enhancing, the level of FXN protein via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful, inter alia, for the treatment of subjects who have, have been diagnosed with having, or are at risk of having a disorder associated with frataxin (FXN) deficiency, e.g., Friedreich's Ataxia (FA), or at least one symptom thereof.