C12N2800/40

VECTORS
20210388384 · 2021-12-16 ·

The present invention provides a kit of vectors comprising a first and second viral vector, wherein the first viral vector comprises a transgene of interest (TOI), and presence of the second viral vector in a host cell is required for integration of the first viral vector TOI into the host cell genome.

ANTI-TUMOR COMPOSITION
20210386806 · 2021-12-16 ·

The present invention relates to an oncolytic adenovirus capable of co-expressing interleukin-12 and a C-met-inhibiting oligonucleotide; and an antitumor immune-boosting composition and anticancer composition comprising the oncolytic adenovirus. The present invention has first identified an adenovirus system having simultaneous effects of IL-12 expression and C-met inhibition in cancer gene treatment. The adenovirus system of the present invention is capable of inhibiting C-met while expressing interleukin-12, thereby restoring immune functions in a tumor environment to enhance anticancer effects such as the inhibition of tumor recurrence and tumor growth and to inhibit tumor migration. Accordingly, the adenovirus system of the present invention can be effectively used in the treatment of cancer.

LONG-LASTING ANALGESIA VIA TARGETED IN VIVO EPIGENETIC REPRESSION
20220202957 · 2022-06-30 ·

The disclosure provides epigenetic based approaches and methods using genome editing constructs comprising a zinc finger fused with a repressor domain and/or a dCas9 fused with a repressor domain to treat and manage pain in subjects in need of treatment thereof.

AAV VECTOR-MEDIATED DELETION OF LARGE MUTATIONAL HOTSPOT FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

Disclosed herein are therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use.

Compositions and Methods for Treating Cancer with DuoCARs

Novel therapeutic immunotherapy compositions comprising at least two vectors, each vector encoding a functional CAR, whereby the combination of vectors results in the expression of two or more non-identical binding domains, wherein each vector encoded binding domain(s) are covalently linked to a transmembrane domain and one or more non-identical intracellular signaling motifs are provided herein as well as are methods of use of same in a patient-specific immunotherapy that can be used to treat cancers and other diseases and conditions.

Methods For Polynucleotide Integration Into The Genome Of Bacillus Using Dual Circular Recombinant DNA Constructs And Compositions Thereof

Methods and compositions are provided for integrating genes of interest into the genome of a Bacillus sp. cell without the integration of a selectable marker into said genome. The methods employ a dual circular recombinant DNA system for introduction of a guide RNA/Cas endonuclease system (also referred to as an RNA guided endonuclease, RGEN) as well as a donor DNA into a Bacillus sp. cell, and providing a highly effective system for inserting genes of interest into the genome of said Bacillus sp. cell.

METHODS AND COMPOSITIONS FOR CORRECTION OF DMD MUTATIONS
20230272428 · 2023-08-31 ·

Disclosed herein are methods and compositions for treating muscular dystrophies, including DMD, by deleting and optionally replacing disease associated mutations in the genome of muscular dystrophy patients.

Production of lytic phages

The present invention concerns a production bacterial cell for producing lytic phage particles or lytic phage-derived delivery vehicles, said production bacterial cell stably comprising at least one phage structural genes and at least one phage DNA packaging genes, said phage structural gene(s) and phage DNA packaging gene(s) being derived from a lytic bacteriophage, wherein the expression of at least one of said phage structural genes and/or at least one of said phage DNA packaging gene(s) in said production bacterial cell is controlled by an induction mechanism.

BIDIRECTIONAL MULTI-ENZYMATIC SCAFFOLDS FOR BIOSYNTHESIZING CANNABINOIDS
20230265465 · 2023-08-24 ·

This document relates to using bidirectional, multi-enzymatic scaffolds to biosynthesize cannabinoids in recombinant hosts.

COMPOSITIONS AND METHODS COMPRISING ENGINEERED CHIMERIC ANTIGEN RECEPTOR AND MODULATOR OF CAR
20220145325 · 2022-05-12 ·

There is provided method for making a cell composition which comprises step of transducing a population of cells with a mixture of at least two viral vectors, wherein at least one vector comprises a nucleic acid sequence which encodes a chimeric antigen receptor (CAR); and wherein at least one vector comprises a nucleic acid encoding an activity modulator which modulates the activity of the CAR, of a cell expressing the CAR, or of a target cell. There is also provided a cell composition made by such a method and its use in the treatment of diseases such as cancer.