13 results about "Stop codon" patented technology

Aspects of the disclosure relate to methods, uses, compositions, kits, and systems for editing an endogenous tRNA into a suppressor tRNA. Additional aspects relate to compositions comprising the prime editing machinery, pegRNAs, and / or complexes comprising the prime editor and pegRNA that are capable of editing and / or replacing an endogenous tRNA to yield a suppressor tRNA. Polynucleotides, cells, vectors, and complexes are also contemplated.

The present disclosure includes bioorthogonal conjugation systems and methods of producing same. The systems include at least one ncAA that participates in an orthogonal click reaction owing to its reactive group. The system further includes at least one plasmid having a gene for one or more of: an aminoacyl-tRNA synthetase, a tRNA, and a nanobody. Each nanobody includes suppression stop codons that define locations for conjugation of a ncAA, such that reactive groups of the ncAAs are incorporated at each defined location of the nanobody. Locations are not restricted to N and C terminals of the peptide, facilitating nanobody functionality post-conjugation. Resulting nanobodies are modified with the reactive groups of the ncAA in one or more locations, such that multiple cargos can be conjugated to each nanobody. Additionally, nanobodies can be conjugated to other nanobodies having corresponding reactive groups to form assemblies, which may be used as therapeutic nanobody libraries.

The application of OsFabF gene in resistance to rice stripe virus, the application relates to the technical field of rice disease resistance breeding. The application uses a CRISPR / Cas9 gene editing system to design sgRNA targeting a specific functional region of an endogenous OsFabF gene in rice, to precisely edit the region, thereby obtaining a mutant with a functionally deficient OsFabF. The editing preferably results in a frame shift mutation or a premature stop codon, to obtain a truncated OsFabF protein mutant. Experiments show that the rice mutant plant obtained in this way exhibits significantly enhanced resistance to RSV infection, reduced virus accumulation and reduced disease symptoms. The application provides an effective gene target and breeding material for cultivating new rice varieties resistant to rice stripe virus.

This invention relates to a mouse model of Parkinson's disease and its applications. The construction method includes the following steps: 1) in mice... Snack An exogenous fragment containing the human SNCA G51D coding sequence was knocked into the region upstream of the gene stop codon via homologous recombination, causing the human SNCA G51D coding sequence to interact with the mouse endogenous gene. Snack The gene forms a bicistronic expression structure, and the coding sequence of the human SNCA G51D is shown in SEQ ID No:1; 2) Positive F0 generation mice are backcrossed with wild-type mice, and genotypes are identified and screened to obtain a stable SNCA G51D endogenous knock-in Parkinson's disease mouse model. The gene editing strategy of the Parkinson's disease mouse model preserves the mouse's... Snack Simultaneous endogenous expression of α-synuclein G51D in the gene can mimic two pathogenic forms, exhibiting high physiological relevance and genetic authenticity.

This invention discloses a method for constructing a Brca2-p.Q462Ter genetically engineered mouse and its application. The Brca2 genetically engineered mouse provided by this invention carries a truncated mutation in its Brca2 gene. This mutation is located at exon 10 of the mouse Brca2-201 transcript, changing amino acid position 462 from glutamine Q (Gln) to the stop codon Ter, forming the Brca2-p.Q462Ter mutation. The Brca2-p.Q462Ter genetically engineered mouse is used to simulate the human BRCA2 gene truncated mutation p.Gln472Ter, c.1414C>T. Amino acid homology analysis shows that the mouse BRCA2 protein Q462 site is homologous to the human BRCA2 protein Q472 site; therefore, this mouse mutation site can be used to simulate the corresponding truncated mutation effect in human BRCA2. This genetically engineered mouse can simulate, at the whole animal level, the abnormal DNA damage repair, impaired homologous recombination repair function, decreased genome stability, and susceptibility to related diseases caused by BRCA2 truncated mutations, thus providing a reliable tool for research on BRCA2-related pathogenesis, developmental effects, drug screening, and efficacy evaluation.

This invention discloses a VIGS vector system based on the RNA virus SsOLV4 and its applications. The application of the RNA virus SsOLV4 in constructing the VIGS vector system is described. The VIGS vector for silencing target genes in *Sclerotinia sclerotiorum* is obtained by inserting a 99 bp target gene fragment in reverse between the CDS stop codon and the 3'UTR of SsOLV4 in the vector pMD18-P1 under the action of homologous recombinase. The vector pMD18-P1 contains the full-length cDNA sequence of the virus SsOLV4. Based on SsOLV4, this invention establishes a VIGS vector, overcoming the complexity, time-consuming nature of *Sclerotinia sclerotiorum* genetic transformation technology and the difficulties caused by the purification of multinucleated *Sclerotinia sclerotiorum* transformants. It can rapidly silence exogenous and endogenous genes in *Sclerotinia sclerotiorum*, making it suitable for rapid and efficient gene silencing and functional studies in *Sclerotinia sclerotiorum*.

The application discloses a kind of for sheep MSTN And SOCS2 The sgRNA of gene cytosine base editing and application thereof, the sgRNA includes nucleotide such as any one or several of SEQ ID NO.1~4.This application is respectively designed sgRNA guide sequence for sheep MSTN And SOCS2 Gene, introduce stop codon in target site using CBE system, select sgRNA guide sequence and its combination from which can efficiently target sheep MSTN And SOCS2 Gene, carry out gene editing efficiency verification on cell, and successfully obtain MSTN And SOCS2 Double gene editing goat by prokaryotic embryo microinjection technology, and gene editing sheep shows muscle fiber thickening, and fast growth rate.This application establishes a set of efficient, precise double gene editing technology system, provides key breeding material and technical support for quickly cultivating muscle developed, fast growth meat sheep new variety.

The application discloses a VIGS carrier for preventing and treating Sclerotinia sclerotiorum and application thereof. The VIGS carrier is obtained by reversely inserting a fragment of a gene which plays an important function in the growth, pathogenicity or sclerotia formation of Sclerotinia sclerotiorum into a CDS stop codon and 3'UTR of a VIGS carrier containing an RNA virus SsOLV4 under the action of a homologous recombinase, the VIGS carrier containing the RNA virus SsOLV4 has Amp resistance, and the full-length cDNA sequence of the RNA virus SsOLV4 has a similarity of greater than or equal to 85% to the sequence shown in SEQ ID NO:1. The application realizes the application of an asymptomatic fungal virus in biological prevention and treatment of plant diseases for the first time, and provides an innovative technical path for green prevention and control of crop sclerotinia.

This invention relates to a gene encoding coagulation factor IX. F9 This study investigates animal models of nonsense mutations, methods for screening hemophilia drugs using these models, and the application of the screened stRNAs in the preparation of hemophilia drugs. Based on an analysis of the probability of nonsense mutations in coagulation factor IX in hemophilia patients, the study verifies that stRNAs can read the coagulation factor IX encoding gene. F9 The function and efficiency of the premature termination codon (PTC) were investigated. Seven Arg-stRNAs were designed to target the coagulation factor IX R75* mutation, and readthrough efficiency was tested at the cellular level. A mouse model of the coagulation factor IX R75* mutation was constructed, and Arg-stRNA was delivered via AAV, restoring the expression of endogenous coagulation factor IX and reducing clotting time, demonstrating the effectiveness of stRNA in treating hemophilia B.

The present application relates to the fields of biotechnology and RNA technology. In particular, the present application relates to an RNA single base editing system based on pseudouridine modification and a method for inhibiting a premature termination codon (PTC) in a target RNA in a host cell. The present application also relates to an RTM, a composition comprising the same, a delivery composition and a host cell. The present application also relates to their use in the preparation of pharmaceuticals and formulations.

Disclosed are universal UTR sequences for mRNA assembly, a composition thereof, and use thereof, belonging to the fields of molecular biology and biotechnology. The sequences are obtained by modifying a 5'UTR sequence and / or a 3'UTR sequence, for example, adding GCCACC at position -1 or adding GCCACC at position -4 of a consensus sequence of the 5'UTR, removing a uORF from the 5'UTR, and adding three sets of stop codons at the 5' end. The provided universal modified 5'UTR and 3'UTR sequences can effectively promote the stability and protein expression level of mRNAs, and are particularly suitable for the fields of gene therapy and therapeutic protein production.

The application discloses a deletion mutant of a rice LHCB5 gene and application thereof, and belongs to the field of gene editing. Through gene editing system, the 3'UTR region of the rice LHCB5 gene is precisely edited, and a 40bp homozygous deletion mutant of +16 to +55bp downstream of the termination codon TAA is obtained. The mutant can be stably inherited, and the growth phenotype is not significantly different from that of the wild type. Meanwhile, the resistance to rice blast and bacterial leaf blight is significantly improved, and the length of the disease spot is significantly reduced. The application first discloses the disease resistance regulation function of the 3'UTR of the LHCB5 gene, and realizes the improvement of the disease resistance through the editing of the non-coding region. No exogenous transgene residue is left, and the safety and agronomic trait risks brought by transgene and coding region editing are avoided. The obtained mutant can be directly used for the breeding of rice broad-spectrum disease resistance, and has important application value.

The disclosure relates generally to suppressor tRNAs engineered to enhance suppression of premature termination and their use in treatment of disorders associated with a premature terminal codon, such as muscular dystrophy and cardiology disorders.