Patent classifications
C12N2830/00
COMBINATORIAL CANCER IMMUNOTHERAPY
Provided herein are methods and compositions for dynamically controlling and targeting multiple immunosuppressive mechanisms in cancer. Some aspects provide cells engineered to produce multiple effector molecules, each of which modulates a different immunosuppressive mechanisms of a tumor, as well as methods of using the cells to treat cancer, such as ovarian, breast, or colon cancer.
Reverse genetics systems
The invention provides various reverse genetics systems for producing segmented RNA viruses, wherein the systems do not require bacteria for propagation of all of their expression constructs.
USE OF ADENOVIRUS AND NUCLEIC ACIDS CODING THEREFOR
This invention relates to the use of an adenovirus to treat cancer, for example. The adenovirus may be replication deficient in cells that lack Y box binding protein. The adenovirus may encode an oncogene or an oncogene product, which may transactivate at least one viral gene.
GENOMIC ENGINEERING OF PLURIPOTENT CELLS
Provided are methods and compositions for obtaining genome-engineered iPSCs, and derivative cells with stable and functional genome editing at selected sites. Also provided are cell populations or clonal cell lines derived from genome-engineered iPSCs, which comprise targeted integration of one or more exogenous polynucleotides, and/or in/dels in one or more selected endogenous genes.
RIBOSWITCH MODULATED GENE THERAPY FOR RETINAL DISEASES
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.
Corynebacterium Constitutive Expression Vector Promoter Screened On The Basis Of Transcriptome Sequencing, Screening Method Thereof, And Applications Thereof
Provided is a method for screening a corynebacterium constitutive expression vector promoter on the basis of transcriptome sequencing; and further provided are the corynebacterium constitutive expression vector promoter screened on the basis of transcriptome sequencing, an expression vector comprising the promoter, a recombination strain obtained by transforming a host cell Corynebacterium glutamicum using the expression vector, and applications thereof.
Use of adenovirus and nucleic acids coding therefor
This invention relates to the use of an adenovirus to treat cancer, for example. The adenovirus may be replication deficient in cells that lack Y box binding protein. The adenovirus may encode an oncogene or an oncogene product, which may transactivate at least one viral gene.
Targeted mutations
The present invention relates to a process for producing and selecting for targeted mutations in bacterial genomes. In particular, the process relates to the transformation of bacteria with a Recombination Element which comprises the desired mutation followed by homologous recombination of the Recombination Element into the bacterial genome; the CRISPR/Cas system is then used to eliminate bacteria which do not have the desired mutation.
REGULATORY SYSTEM FOR EXPRESSION OF A GENE OF INTEREST IN A TARGET CELL AND METHOD OF USE THEREOF
Provided is a system for regulating expression of a gene of interest in a target cell, including a recombinant first RNA molecule with (i) a coding sequence for a translation-suppressor protein and (ii) a first microRNA (miR) recognition element in its 3 UTR, wherein the first miR recognition element recognizes a first miR and binding of a first miR to the first miR recognition element reduces translation of the translation suppressor, and a recombinant second RNA molecule, with (i) a coding sequence for the gene of interest, (ii) a recognition sequence for the translation-suppressor, wherein binding of the translation-suppressor to the recognition sequence for the translation-suppressor reduces translation of the gene of interest, and, optionally, (iii) a second miR recognition element in its 3 UTR, wherein the second miR recognition element recognizes one or more second miR and binding of one or more of the one or more second miR to the second miR recognition element reduces translation of the gene of interest. Also provided are methods of using the system.
SYNERGISTIC EFFECT OF SMN1 AND MIR-23A IN TREATING SPINAL MUSCULAR ATROPHY
The present application relates to the fields of biotechnology, virology, genetics, and molecular biology. More specifically, the present invention relates to an isolated nucleic acid for producing a gene therapy viral product, said isolated nucleic acid comprising a nucleic acid that encodes the SMN1 protein having the amino acid sequence of SEQ ID NO: 1, and a nucleic acid that encodes the microRNA miR-23a, an expression cassette and a vector based thereon, as well as an AAV9-based recombinant virus for expressing the SMN1 gene in target cells, a pharmaceutical composition that includes said recombinant virus, and various uses of the above recombinant virus and the above composition.