C12N2740/15071

PERSISTENT MEMORY T-CELL RESPONSES TO CANCER AND INFECTIOUS-DISEASE ANTIGENS BY MANIPULATION OF AMINO ACID-CATABOLISM PATHWAYS

Provided herein are recombinant polynucleotides including a first nucleic acid sequence encoding an antigen, and a second nucleic acid sequence encoding an enzyme of an amino acid catabolic pathway. The provided recombinant polynucleotides are particularly useful for inducing long-lived immune responses having improved memory characteristics. Also provided are pharmaceutical compositions, viral particles, and host cells including the disclosed recombinant polynucleotides, and methods for using the disclosed materials.

Vectors and compositions for treating hemoglobinopathies

The invention provides improved gene therapy vectors, compositions, and methods.

NIPAH VIRUS ENVELOPE PSEUDOTYPED LENTIVIRUSES AND METHODS OF THEIR USE

The present invention relates to lentiviral particles which have been pseudotyped with Nipah virus (NiV) fusion (F) and attachment (G) glycoproteins (NiVpp-F/G). Additionally, the present invention relates to truncated NiV-F glyocproteins useful in producing such NiVpp lentiviral particles, as well as to additional variant peptides which enhance activity. Further, the present invention relates to methods of using such lentiviral particles or sequences, for example in the treatment of cancer or CNS disorders.

Systemic and local ex vivo gene therapy of the skeleton

Methods are disclosed herein for increasing bone mass and strength or bone fracture healing in a subject. The methods include administering to the subject a therapeutically effective amount of multipotent stem cells, wherein each multipotent stem cell is transformed with a recombinant nucleic acid molecule comprising a heterologous promoter operably linked to a nucleic acid encoding platelet derived growth factor (PDGF) B, and wherein the multipotent stem cells express a sufficient amount of PDGFB to increase bone mass and strength or bone fracture healing. A lentiviral vector also is disclosed that includes a phosphoglycerate kinase-1 (PGK) promoter operably linked to a nucleic acid encoding PDGFB.

Replication-competent adenoviral vectors

This invention provides improved replication-competent adenoviral vectors. The improved vectors have both a hybrid regulatory unit that provides for high level transgene expression. The vectors can be use, e.g., for therapeutic or prophylactic purposes.

TRANSGENIC VERO-CD4/CCR5 CELL LINE
20170266272 · 2017-09-21 ·

The present relation relates to a transgenic Vero cell line expressing CD4 and CCR5. The present invention encompasses the preparation and purification of immunogenic compositions which are formulated into the vaccines of the present invention.

CMV Glycoproteins and Recombinant Vectors
20210395771 · 2021-12-23 ·

Disclosed herein are recombinant CMV vectors which may comprise a heterologous antigen that can repeatedly infect an organism while inducing a CD8+ T cell response to immunodominant epitopes of the heterologous antigen. The CMV vector may comprise a deleterious mutation in the US11 glycoprotein or a homolog thereof.

COMPOSITIONS AND METHODS FOR TREATMENT OF HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS

Provided herein are nucleic acids, vectors, and cells containing an expression-optimized codon that encodes a Munc13-4 polypeptide or a STXBP2 polypeptide. Also provided are methods of making and using the nucleic acids, vectors, and cells. Also provided herein are methods of treating of Hemophagocytic Lymphohistiocytosis (HLH) in a subject.

Specific chimeric antigen receptor cells targeting human CLDN18A2, preparation method and application thereof

A chimeric antigen receptor (CAR) cell specifically targeting human Claudin18.2 (CLDN18A2), a preparation method and an application thereof are provided. The extracellular binding region of the CAR includes a protein specifically recognizing CLDN18A2 that has any one of the amino acid sequences as shown in SEQ ID NOS: 2-5 or any one of the amino acid sequences of the variants having 70%-99% identity with the amino acid sequences shown by the SEQ ID NOS: 2-5. The immune effector cell modified by the CAR can be used to treat tumors.

COMPOSITIONS AND METHODS FOR IN VIVO GENERATION OF CAR EXPRESSING CELLS

Aspects of this disclosure relate generally to the use of biomaterials for the in vivo generation of CAR expressing cells. In some embodiments, the biomaterials comprise one or more of a cell recruitment composition, a viral vector, and/or a cell activation agent.