C12N2830/50

IMPROVED HVT-VECTORED ND-IBD VACCINE

The present invention regards a new and Improved HVT-vectored ND-IBD vaccine, comprising a recombinant HVT comprising the VP2 gene from IBDV and the F gene from NDV to a target animal. The recombinant HVT can be used in a vaccine for poultry, which displayed good viral vector replication, effective expression of the NDV F—and IBDV VP2 genes, improved immunoprotection against ND and IBD, and improved genetic stability over prior art constructs.

REGULATABLE ADENO-ASSOCIATED VIRUS (AAV) VECTOR
20170304464 · 2017-10-26 ·

The present invention relates to regulatable adeno-associated virus (AAV) vectors as well as to their use in gene therapy. It further relates to corresponding nucleic acid molecules, host cells, non-human transgenic animals, pharmaceutical compositions and kits.

ADENO-ASSOCIATED VIRUS VECTORS FOR TREATMENT OF RETT SYNDROME
20220056478 · 2022-02-24 ·

The disclosure provides nucleic acids (comprising AAV expression cassettes), AAV vectors, and compositions for use in methods for treating and/or delaying the onset of diseases associated with mutations in the mecp2 gene, such as Rett Syndrome. Also, provided herein are methods for treating and/or delaying the onset of brain-derived neurotrophic factor (BDNF)-associated diseases.

ACE2-Targeted Compositions and Methods for Treating COVID-19
20220056153 · 2022-02-24 · ·

This invention provides a monoclonal antibody that (i) specifically binds to the extracellular portion of human angiotensin converting enzyme 2 (hACE2); (ii) specifically inhibits binding of SARS-CoV-2 to the extracellular portion of hACE2; and (iii) does not significantly inhibit the ability of hACE2 to cleave angiotensin II and/or a synthetic MCA-based peptide. This invention also provides related recombinant AAV vectors, recombinant AAV particles, compositions, prophylactic and therapeutic methods, and kits.

Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy

The present invention provides compositions comprising retroviral vectors, transduced cells, and methods of using the same for gene therapy. In particular, the present invention relates to lentiviral vectors and cells transduced with those vectors to provide gene therapy to subjects having an adrenoleukodystrophy and/or adrenomyeloneuropathy.

Adeno-Associated Viral Vectors for the Gene Therapy of Metabolic Diseases

The present invention discloses adeno-associated viral vectors useful in gene therapy methods for the treatment of obesity, insulin resistance, type 2 diabetes, liver cirrhosis and non-alcoholic fatty liver disease (NAFLD)/non-alcoholic steatohepatitis (NASH). The invention also relates to polynucleotides, plasmids, vectors and methods for the production of such adeno-associated viral vectors. The invention also relates to pharmaceutical compositions comprising said vectors.

TRANSGENE CASSETTES, AAV VECTORS, AND AAV VIRAL VECTORS FOR EXPRESSION OF HUMAN CODON-OPTIMIZED CSTB

The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to CSTB loss and/or misfunction. The methods and compositions of the present disclosure include rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules encoding CSTB polypeptides.

SARS-COV2 VACCINE VECTOR METHODS AND COMPOSITIONS
20220048954 · 2022-02-17 ·

Provided herein are recombinant nucleic acids and viral vectors thereof encoding a SARS-CoV-2 spike protein that have been optimized for expression in mammalian cells.

Methods and compositions for controlling gene expression by RNA processing

The present disclosure provides nucleic acids encoding an RNA recognition sequence positioned proximal to an insertion site for the insertion of a sequence of interest; and host cells genetically modified with the nucleic acids. The present disclosure also provides methods of modifying the activity of a target RNA, and kits and compositions for carrying out the methods.

Viral vector

Provided is a multicistronic retroviral vector genome having a first nucleic acid sequence upstream of at least one internal regulatory element, such that the level of genomic RNA available for packaging in the absence of rev, or a functional equivalent thereof, is increased.