6258results about "Reverse transcribing RNA viruses" patented technology

To provide methods for treating a subject having or at risk of developing a neurocognitive disorder, such as frontotemporal lobar degeneration or neuronal ceroid lipofuscinosis.SOLUTION: Provided is a method comprising administering, to a subject diagnosed as having a neurocognitive disorder (NCD), a composition comprising a population of cells comprising a transgene encoding a progranulin (PGRN) or a granulin (GRN).SELECTED DRAWING: Figure 1A

Compositions of retroviruses and methods of using the same for gene delivery are disclosed, wherein the retroviruses comprise a viral envelope protein comprising at least one mutation that diminishes its native function, a non-viral membrane-bound protein comprising a membrane-bound domain and an extracellular targeting domain.

The present disclosure provides compositions and methods comprising recombinant particles suitable for specifically delivering one or more engineered receptors to immune effector cells in vivo.

Described herein are systems and methods for automated cell transduction within a cell processing system. A system for cell processing may include a cell processing cartridge having a transduction system. The transduction system may include a fluidic manifold, one or more modules for performing a cell processing protocol, and a tube having a surface area to volume ration of between about 1,260 mm2 / mL and about 5,080 mm2 / mL. A method for cell processing may first include flowing cells through a tube of a flow cell of a cell processing cartridge for a first time period to achieve a transduction efficiency of at least 50%. Second, the method may include expanding the cells within a bioreactor module of the cell processing cartridge for a second time period.

Provided herein are gene editing pairs comprising an engineered protein, e.g, an engineered CRISPR protein and a guide ribonucleic acid (gRNA) that may provide improved gene editing relative to reference, i.e., wild type sequences. In some embodiments, the engineered protein of the gene editing pair comprises a RuvC cleavage domain with one or more amino acid modifications relative to a reference CRISPR protein RuvC sequence. In some embodiments, a guide ribonucleic acid (gRNA) of the gene editing pair comprises at least one modification in a scaffold stem loop relative to a reference scaffold stem loop sequence.

The present invention provides a method for packaging a BaEV retroviral vector. The method includes constructing a stable BaEV retroviral packaging cell line, transfecting a retroviral vector plasmid containing a target sequence into a virus-producing cell line, where the virus-producing cell line is a HEK293T cell line or its derivative cell line, to harvest the transiently transfected and virus-producing retroviral vector, mixing it with the stable BaEV retroviral packaging cell line, and promoting the transduction of the retroviral vector into the stable BaEV retroviral packaging cell line by horizontal centrifugation. The present invention can achieve the packaging of a BAEV-type retroviral vector carrying a CD19CAR foreign gene plasmid, construct a BAEV-type retroviral vector-producing cell line, and produce a high-titer BAEV-type retroviral vector.

The invention provides an UTR (Untranslated Region) element H2202 P1-G as well as a construction method and application thereof, and relates to the technical field of mRNA (messenger ribonucleic acid). According to the present invention, the ribosome load prediction and the secondary structure optimization are performed on the natural 5 'UTR of the HIV TAT 202 gene through the BaidleHelix platform, and the obtained HTAT 202 P1 sequence avoids the inhibitory hairpin structure so as to significantly improve the luciferase expression quantity compared to the natural UTR; an ncRNA sequence without a secondary structure is introduced on the basis of the HTAT 202 P1, translation inhibition of a 5 'cap region is further relieved, and the protein expression quantity of the constructed H2202 P1-G mutant (the DNA sequence of the H2202 P1-G is as shown in SEQ NO 1, and the RNA sequence is as shown in SEQ NO 2) is further improved.

Provided herein are lentiviral vectors comprising a mutated, heterologous envelope protein, a targeting protein, and at least one transgene for delivery to and expression by a cell characterized by the targeting protein. Also provided are methods and materials for producing the lentiviral vectors described herein, methods for transducing target cells, and cells transduced by lentiviral vectors according to the present disclosure.

The invention discloses an oligopeptide composition for inhibiting inclusion body formation and promoting Gag-Like protein nanoparticle self-assembly, the oligopeptide composition comprises a C-terminal oligopeptide and an N-terminal oligopeptide, the amino acid sequence of the N-terminal oligopeptide is as shown in SEQ ID NO: 1, and the amino acid sequence of the C-terminal oligopeptide is as shown in SEQ ID NO: 2. The oligopeptide composition can effectively avoid formation of a prokaryotic expression protein inclusion body and / or promote dissolution of prokaryotic expression protein, the oligopeptide composition is further utilized to be combined with HIV-1p24 and human endogenous retrovirus K capsid protein CA, nanoparticles are prepared respectively, and the oligopeptide composition can be used as a carrier for antigen surface display or drug transportation. The nanoparticles formed by the HIV-1 capsid protein p24 and the human HERV K CA have cavities which are obviously larger than those of ferritin, so that the nanoparticles have higher loading capacity; as an antigen display platform, the antibody also has good stability and wide applicability.

Disclosed herein are compositions and methods for modulating T cells. For example, the compositions and methods may be used to increase memory T cells. The compositions and methods may increase the expression or protein level of a transcription factor selected from THAP6, DMRT3, MEF2B, PAX2, ATOH7, KLF2, KLF1, TWIST1, NKX6, and FEV, or a combination thereof. The compositions and method may be used in combination with Adoptive T Cell Therapy (ACT) to enhance the ACT.

The invention provides an enhancer RNA (Ribonucleic Acid) molecule MZGAe1 and application thereof, and relates to the technical field of biology. The invention provides an enhancer RNA (Ribonucleic Acid) molecule MZGAe1. The nucleotide sequence of the enhancer RNA molecule MZGAe1 is as shown in SEQ ID NO: 1; meanwhile, the invention further provides a specific sgRNA sequence for activating the molecule in a targeted manner and a recombinant vector of the specific sgRNA sequence. Expression of endogenous MZGAe1 of cells is specifically activated by adopting a CRISPR activation technology, and conversion of mouse embryonic stem cells to bicellular-like cells can be efficiently promoted. By providing a brand-new endogenous targeted MZGAe1 accurate activation tool, the efficient transformation of wild mouse embryonic stem cells to bicellular cells can be realized only under the condition of endogenous activation of single enhancer RNA molecule MZGAe1, the proportion is at least increased by 5%, the expression of a totipotent marker gene ZSCAN4 is activated, and the expression of the totipotent marker gene ZSCAN4 is promoted. And an efficient and specific brand-new research tool and scheme are provided for researching zygote genome activation, embryonic development early events and cell reprogramming.

Disclosed herein are compositions, methods, kits, and systems relating to efficient delivery of cargos (e.g., therapeutic cargos) into cells, for instance, for in vivo delivery. The present disclosure provides lipid-containing particles (e.g., virus-like particles) for delivering therapeutic cargos. The present disclosure also provides polynucleotides encoding the lipid-containing particles provided herein, which may be useful for producing said lipid-containing particles. Also provided are methods for editing nucleic acid molecules in cells using the lipid-containing particles provided herein, as well as cells and kits comprising the lipid-containing particles.

The invention discloses a construction method and application of a gene modified mesenchymal stem cell. The construction method comprises the following steps: constructing a recombinant lentivirus carrying PD-L1 and ICAM1 genes; the recombinant lentivirus carrying the PD-L1 gene and the ICAM1 gene is transfected with mesenchymal stem cells, and the mesenchymal stem cells are screened by puromycin, so that an engineered mesenchymal stem cell strain modified by the PD-L1 gene and the ICAM1 gene is obtained; the mesenchymal stem cells provided by the invention are modified by engineering genes of PD-L1 and ICAM1, and are screened by puromycin to obtain a mesenchymal stem cell strain capable of stably and highly expressing the genes of PD-L1 and ICAM1; after the mesenchymal stem cells highly expressing the PD-L1 and ICAM1 genes are intravenously injected into an autoimmune hepatitis animal model, the autoimmune hepatitis symptom can be remarkably relieved, the liver injury degree can be reduced, the expression of inflammatory factors can be regulated, the inflammatory reaction can be inhibited, and the obvious curative effect can be achieved.

The present disclosure provides compositions and methods comprising recombinant particles suitable for specifically delivering one or more chimeric antigen receptors to immune effector cells in vivo.

The present disclosure provides compositions comprising antigen binding polypeptides. More particularly, the disclosure relates to polypeptides comprising antibodies or antigen binding fragments thereof, nucleic acids encoding the polypeptides, and vectors for expressing the same.

The present disclosure provides compositions comprising antigen binding polypeptides. More particularly, the disclosure relates to polypeptides comprising antibodies or antigen binding fragments thereof, nucleic acids encoding the polypeptides, and vectors for expressing the same.

The present disclosure provides compositions comprising antigen binding polypeptides. More particularly, the disclosure relates to polypeptides comprising antibodies or antigen binding fragments thereof, nucleic acids encoding the polypeptides, and vectors for expressing the same.

The invention discloses a hybridoma cell strain, a monoclonal antibody, a linear epitope antigen and application thereof, and relates to the field of genetic engineering, in particular to a hybridoma cell strain, a monoclonal antibody, a linear epitope antigen and application thereof. The preservation number of the hybridoma cell strain P27-A1 is CGMCC (China General Microbiological Culture Collection Center) No. 46352. The hybridoma cell strain P27-A1 is prepared from feline leukemia virus p27, and a monoclonal antibody secreted by the hybridoma cell strain P27-A1 recognizes a linear epitope antigen of feline leukemia virus p27 protein; the kit for the feline leukemia virus p27 comprises the monoclonal antibody A1 secreted by the hybridoma cell strain P27-A1. The monoclonal antibody secreted by the hybridoma cell strain can greatly improve the sensitivity of the p27 protein for detecting feline leukemia virus. The invention provides the monoclonal antibody secreted by the hybridoma cell strain for identification, and the monoclonal antibody can be used for preparing a diagnostic kit such as a colloidal gold test strip.

The invention relates to the technical field of biological medicine, in particular to a T cell receptor targeting AFP peptide, a preparation method of the T cell receptor and a companion diagnostic kit. A T cell receptor targeting an AFP peptide is provided, the sequence of the AFP peptide is FMNKFIYEI, and the T cell receptor comprises a TCR [alpha] chain variable domain and a TCR [beta] chain variable domain. Corresponding nucleic acid molecules, vectors and host cells, and methods of making T cell receptors are provided. Further provided is a companion diagnostic kit containing the cell receptor. The invention provides a better choice for concomitant diagnosis and treatment products of cells of alpha fetoprotein (AFP).

A method of treating or inhibiting a viral infection in a human subject, such as a SARS-CoV-2 infection, involves inhibiting in vivo the expression or activity of one or a combination of the subject's genes required for viral infection. Single genes or subsets of genes for inhibition of activity or expression are selected from certain identified genes. Methods of administration of certain known small molecules or other therapeutics which mimic loss of function of the identified genes are provided. Similar methods for conducting screens of host genes required for viral infection are shown.

Disclosed herein are compositions of retroviruses and methods of using the same for gene delivery, wherein the retroviruses comprise a viral envelope protein comprising at least one mutation that diminishes its native function, a non-viral membrane-bound protein comprising a membrane-bound domain and an extracellular targeting domain.

The present disclosure provides compositions comprising antigen binding polypeptides. More particularly, the disclosure relates to polypeptides comprising antibodies or antigen binding fragments thereof, nucleic acids encoding the polypeptides, and vectors for expressing the same.

Disclosed herein are compositions, methods, kits, and systems relating to efficient delivery of cargos (e.g., therapeutic cargos) into cells, for instance, for in vivo delivery. The present disclosure provides lipid-containing particles (e.g., virus-like particles) for delivering therapeutic cargos. The present disclosure also provides polynucleotides encoding the lipid-containing particles provided herein, which may be useful for producing said lipid-containing particles. Also provided are methods for editing nucleic acid molecules in cells using the lipid-containing particles provided herein, as well as cells and kits comprising the lipid-containing particles.

The invention provides compositions and methods for manufacturing adoptive cell therapies. In particular embodiments, the invention provides methods of harvesting populations of cells, isolating and activating PBMCs, expanding T cells, and administering the T cell therapeutic to a subject in need thereof.

The use of dual-target CAR-T cells in treating systemic lupus erythematosus and other B-cell autoimmune diseases is provided. The first target of the chimeric antigen receptor is CD19, the second target is BCMA, and specifically, the chimeric antigen receptor is a chimeric antigen receptor with a Loop structure, which is shown as follow: L-VL, CD19-VH, BCMA-VL, BCMA-VH, CD19-H-TM-C-CD3ζ; the BCMA-CD19 CAR-T cells transduced by the chimeric antigen receptor targeting CD19 and BCMA can completely block the pathway of producing autoantibodies, and completely cover the B cells, plasmablasts and plasma cells at multiple differentiation stages that produce autoantibodies, so that the dual-target CAR-T therapy designed based on this concept can be aimed at refractory SLE and is expected to achieve faster remission and deeper and lasting curative effect.

The present disclosure provides virus-like particles (VLPs) for delivering prime editors, and systems comprising such prime editor (PE) VLPs. The present disclosure also provides polynucleotides encoding the PE-VLPs described herein, which may be useful for producing said PE-VLPs. Also provided herein are methods for editing the genome of a target cell by introducing the presently described PE-VLPs into the target cell. The present disclosure also provides fusion proteins that make up a component of the PE-VLPs described herein, as well as polynucleotides, vectors, cells, and kits.

Disclosed herein are compositions, methods, kits, and systems relating to efficient delivery of cargos (e.g., therapeutic cargos) into cells, for instance, for in vivo delivery. The present disclosure provides lipid-containing particles (e.g., virus-like particles) for delivering therapeutic cargos. The present disclosure also provides polynucleotides encoding the lipid-containing particles provided herein, which may be useful for producing said lipid-containing particles. Also provided are methods for editing nucleic acid molecules in cells using the lipid-containing particles provided herein, as well as cells and kits comprising the lipid-containing particles.

The invention provides a lentivirus for treating cancer as well as a preparation method, application and a corresponding pharmaceutical composition of the lentivirus. According to the invention, an hTERT promoter and a CDC6shRNA gene sequence are inserted behind an enzyme cutting site of a vector, an obtained plasmid is transferred into an expression strain, the expression strain is cultured and screened to obtain a cloned strain, and the cloned strain is shaken and packaged to obtain the hTERT-CDC6shRNA lentivirus; when the lentivirus disclosed by the invention is used for infecting cancer cells, the experimental result shows that the proliferation of the cancer cells can be effectively inhibited.

The invention relates to a method of modulating the expression of an endogenous gene in a somatic cell derived from an induced pluripotent stem cell (iPSC), comprising differentiating an engineered iPSC capable of stable or inducible expression of a CRISPR-related protein into a somatic cell prior to introduction of a guide RNA (gRNA) sequence. The invention also relates to the engineered iPSC, cells obtained by the method and uses thereof.