C12N2740/15044

siRNA EXPRESSION VECTOR
20220372516 · 2022-11-24 · ·

Provided is a nucleic acid construct for expressing a gene of interest, the nucleic acid construct comprising, in order from 5′-terminus, each sequence of: (a) a 5′ long terminal repeat (LTR) sequence derived from a retrovirus; (b) a packaging signal sequence (IV) derived from a retrovirus; (c) a sequence or multiple cloning site of the gene of interest; (d) a post-transcriptional regulatory sequence (PRE); (e) an siRNA generating sequence which forms at least one stem-loop structure and in which RNA, which induces RNA interference in mammalian cells, is transcribed; and (f) a 3′ LTR sequence derived form a retrovirus.

VIRAL TARGETING OF HEMATOPOIETIC STEM CELLS

Disclosed herein are compositions of retroviruses and methods of using the same for gene delivery to a hematopoietic stem cell (HSC), 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.

Compositions comprising lentiviral vectors expressing IL-12 and methods of use thereof

This patent application relates generally to the treatment of cancer, and more particularly to the use of a pseudotyped lentivirus expressing IL-12 for the treatment of cancer.

RECOMBINANT BOVINE FOLLICLE-STIMULATING HORMONE, COMPOSITION COMPRISING THE SAME, AND METHOD TO INDUCE SUPEROVULATION AND ESTRUS SYNCHRONIZATION IN BOVINES USING SAID HORMONE

The present invention relates to the technical field of Biotechnology and the obtaining of molecules by recombinant DNA technology, in particular it refers to a synthetic polynucleotide sequence encoding a single-chain recombinant bovine follicle-stimulating hormone, comprising a first polynucleotide enconding the beta subunit of the Bos taurus follicle-stimulating hormone having sequence SEQ ID No. 1, or a variant thereof with at least 85% sequence identity therewith; a second polynucleotide encoding the alpha subunit of the Bos taurus follicle-stimulating hormone having sequence SEQ ID No. 2 or a variant thereof with at least 85% sequence identity therewith; and a third polynucleotide linking said first and second polynucleotides, which encodes a spacer sequence derived from human keratin 10 having sequence SEQ ID No. 3 or a variant. The invention also includes the genetic construct including these sequences, the cells transformed therewith, and the pharmaceutical composition comprising said single-chain recombinant bovine follicle-stimulating hormone.

REV-DEPENDENT LENTIVIRAL VACCINE PARTICLES FOR REDUCING VIRAL REBOUND AND VIRAL RESERVOIRS IN VIVO
20200345833 · 2020-11-05 ·

Persistence of HIV in anatomic sanctuary sites such as the brain prevents viral eradication. Although combination antiretroviral therapy (cART) inhibits viral replication to undetectable level by standard clinical assay, it does not selectively eliminate virus reservoirs. To target HIV reservoirs, the present inventor developed an HIV Rev-dependent lentiviral vector carrying a series of therapeutic genes, such as diphtheria toxin, anthrolysin O from Bacillus anthracis, human TRAF6, or the herpes simplex 1 virus thymidine kinase gene (HSV-tk). The present disclosure provides the Rev-dependent vectors for targeting viral reservoir in a SIV/rhesus macaque model. SIV-infected rhesus macaques were first treated with cART for over 6 months starting 12 weeks post infection, followed by injections with viral particles assembled from a SIV Rev-dependent vector carrying HSV-tk. Following particle injection, animals were further treated briefly (two weeks) with ganciclovir (GCV), which induces the killing of SIV+, HSV-tk expressing cells. cART was terminated following the GCV treatment, and there was observed a partial control of viral rebound over a period of 4 months after cART cessation. The animal was further treated with additional Rev-dependent vector particles, and viral load was diminished to the undetectable level for over 1 year in the absence of any treatment. These results suggest that the Rev-dependent vector, with or without a functional gene, has the potential to diminish viral reservoirs in vivo and can offer a cure of functional cure of HIV/SIV infection.

STABLE INTEGRATION OF SIN TRANFER VECTORS

The present invention relates to the field of the production of lentiviral vectors (LV) for gene therapy. More particularly, the invention relates to a system to obtain the stable integration of a Self Inactivating (SIN) lentiviral transfer vector into a packaging cell line. The SIN lentiviral transfer vector is integrated using a DNA fragment containing the Inverted Terminal Repeats (ITR) of Adeno Associated Virus (AAV), thus obtaining the stable producer cell line for SIN lentiviral vectors.

MAMMALIAN CELLS AND METHODS FOR ENGINEERING THE SAME

The present disclosure provides mammalian cell lines for expression of viral vectors, and methods of making and using the same. Provided methods employ use of identifiers that are capable of being packaged into a viral vector to select and/or identify mammalian cell lines with engineered sequences associated with beneficial characteristics for viral vectors production. Exemplary viral vectors include AAV vectors.

MAMMALIAN CELLS AND METHODS FOR ENGINEERING THE SAME

The present disclosure provides mammalian cell lines for expression of viral vectors, and methods of making and using the same. Provided methods employ use of identifiers that are capable of being packaged into a viral vector to select and/or identify mammalian cell lines with engineered sequences associated with beneficial characteristics for viral vectors production. Exemplary viral vectors include AAV vectors.

COMPOSITIONS COMPRISING LENTIVIRAL VECTORS EXPRESSING IL-12 AND METHODS OF USE THEREOF

This patent application relates generally to the treatment of cancer, and more particularly to the use of a pseudotyped lentivirus expressing IL-12 for the treatment of cancer.

Mammalian cells and methods for engineering the same

The present disclosure provides mammalian cell lines for expression of viral vectors, and methods of making and using the same. Provided methods employ use of identifiers that are capable of being packaged into a viral vector to select and/or identify mammalian cell lines with engineered sequences associated with beneficial characteristics for viral vectors production. Exemplary viral vectors include AAV vectors.