C12N2830/003

Modified adenoviruses

Compositions include modified adenoviruses. Nucleotides, cells, and methods associated with the compositions, including their use as vaccines. Viral vectors using a TET promoter system and methods of producing viruses having the same.

CO-REGULATORY SEQUENCES BASED ON TETRACYCLINE AND CUMATE
20220364103 · 2022-11-17 ·

The present disclosure provides a nucleic acid sequence for regulating the transcription of a nucleic acid fragment of interest, wherein the nucleic acid sequence comprises at least 2 copies of TetO-operator sequences capable of binding to a transactivator rtTA regulatable by tetracycline or a derivative thereof, and 1 copy of minimal promoter sequence containing a TATA box sequence, and at least 1 copy of a CuO-operator sequence capable of binding to a transcription repressor CymR regulatable by cumate. The present disclosure also provides a vector and a host cell containing the nucleic acid sequence, and a method for inducing the expression of a nucleic acid fragment of interest in a host cell.

DIRECT REPROGRAMMING OF SOMATIC CELLS INTO MYOGENIC CELLS

Described herein are methods of generating induced muscle progenitor cells (iMPCs) and uses thereof. Embodiments further provide for methods of promoting muscle regeneration and/or repair and methods of treating a muscle disease or disorder.

Methods for screening variant of target gene

Provided are methods for screening a desired variant of a target gene in a eukaryotic system. Compositions for screening a desired variant of a target gene are also provided.

Methods for reprogramming somatic cells

The invention provides methods for reprogramming somatic cells to generate multipotent or pluripotent cells. Such methods are useful for a variety of purposes, including treating or preventing a medical condition in an individual. The invention further provides methods for identifying an agent that reprograms somatic cells to a less differentiated state.

ACTIVATION OF LYTIC GENES IN CANCER CELLS

The present disclosure provides methods of inducing EBV early lytic cycle genes with high specificity. These methods slow or stop cancer cell growth in vitro and in vivo.

METHODS FOR SCREENING VARIANT OF TARGET GENE
20230109885 · 2023-04-13 ·

Provided are methods for screening a desired variant of a target gene in a eukaryotic system. Compositions for screening a desired variant of a target gene are also provided.

DETECTION OF GENE LOCI WITH POLYCHROMATIC CRISPR-ASSOCIATED PROTEIN 9

A C9orf72 DNA repeat expansion can be detected using a CRISPR Arrayed Repeat Detection System (CARDS). Based upon the compositions and methods supporting this platform primary cell cultures and/or blood cell smears can be tested under conventional clinical diagnostic laboratory conditions to diagnose genetically-based diseases having DNA repeat expansions, including but not limited to ALS. dCas9 constructs are also contemplated as having fluorescent proteins bound to any or all stem loop sequences, wherein detection of a plurality of dCas9 constructs having different colored fluorescent proteins can simultaneously detect at least six (6) different gene target loci.

Inducible expression from transposon-based vectors and uses

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.

IN VIVO METHODS FOR IDENTIFYING CANCER-ASSOCIATED IMMUNOTHERAPY TARGETS

The invention provides in vivo methods for identifying cancer-associated immunotherapy targets.