Methods and composition for modulating a target genome and stable integration of a transgene of interest into the genome of a cell are disclosed.

The present invention refers to hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB). The invention further refers to corresponding nucleic acids producing these variants, to a gene transfer system for stably introducing nucleic acid(s) into the DNA of a cell by using these hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB) and to transposons used in the inventive gene transfer system, comprising a nucleic acid sequence with flanking repeats (IRs and/or RSDs). Furthermore, applications of these transposase variants, the transposon, or the gene transfer system are also disclosed such as gene therapy, insertional mutagenesis, gene discovery (including genome mapping), mobilization of genes, library screening, or functional analysis of genomes in vivo and in vitro. Finally, pharmaceutical compositions and kits are also encompassed.

Provided are a chimeric antigen receptor that targets B-cell maturation antigen (BCMA), and a use thereof, wherein immune cells expressing the chimeric antigen receptor can be effectively used in the treatment and prevention of B cell-related diseases, particularly multiple myeloma or non-Hodgkin's lymphoma.

A system for batch production of the heterogametic sex of a biological species generally includes a first strain of a biological species genetically engineered to include a conditional Y-linked (or Z-linked) genetic lethal circuit and a second strain of the biological species genetically engineered to include a conditional X-linked (or W-linked) genetic lethal circuit.

The present disclosure is directed to compositions and methods for the treatment of Metabolic Liver Disorders. The compositions and methods can comprise an adeno-associated virus (AAV) piggyBac polynucleotide comprising a transgene. The transgene may comprise ornithine transcarbamylase (OTC) or methylmalonyl-CoA mutase (MUT1).

One variation of a method includes: during an initial period, modifying a population of insects to produce a target compound responsive to application of a stressor; during a growth period succeeding the initial period, cultivating the population of insects according to a set of growth conditions; and, during a treatment period succeeding the growth period, applying a dosage of the stressor to the population of insects to trigger production of the target compound. The method further includes, during a harvest period succeeding the treatment period: harvesting the population of insects; homogenizing the population of insects to form a blend including a set of secondary components and an amount of the target compound; extracting the amount of the target compound from the blend; and mixing the first amount of the first target compound with a set of stabilizing agents configured to stabilize the target compound.

Provided are engineered transposable elements, gene transfer systems comprising the engineered transposable elements, as well as methods and kits for using the same. The compositions, systems, and methods are useful for inserting a heterologous nucleic acid into a target nucleic acid in vitro or in a cell.

Methods and composition for modulating a target genome and stable integration of a transgene of interest into the genome of a cell are disclosed.

This disclosure relates to an efficient protein expression system that utilizes piggyBac transposons and/or regulatory elements.

Recombinant expression vectors are disclosed that include a control sequence for recombinant expression of proteins of interest; the control sequence combines a mCMV enhancer sequence with a rat EF-1alpha intron sequence. Some of the vectors are useful for tetracycline-inducible expression. Some of the vectors contain a 5′ PiggyBac ITR and a 3′ PiggyBac ITR to promote genomic integration into a host cell chromosome. A method of selecting a stable production cell line for manufacturing a protein of interest is also disclosed. Also disclosed are mammalian host cells comprising the inventive recombinant expression vectors and a method of producing a protein of interest, in vitro, involving the mammalian host cell.