Patent classifications
C12N2740/15071
FUSOGENIC RHABDOVIRUS GLYCOPROTEINS AND USES THEREOF
Provided for herein are fusogenic rhabdovirus glycoproteins and uses thereof, compositions comprising the same, and methods of using the same. Also provided for herein are pseudotyped viral particles comprising rhabdovirus glycoproteins as provided for herein and targeting moieties as provided for herein. Also provided are methods of generating and using the pseudotyped viral particles as provided for herein.
TARGETED LIPID PARTICLES AND COMPOSITIONS AND USES THEREOF
Provided herein are lipid particles containing a lipid bilayer enclosing a lumen or cavity, a henipavirus F protein molecule or biologically active portion thereof, and a targeted envelope protein containing a henipavirus envelope attachment glycoprotein G (G protein) or biologically active portion thereof and a binding domain, such as a single domain antibody (sdAb) variable domain. Also provided herein are targeted envelope proteins containing a G protein fused or linked to a binding domain, such as a sdAb variable domain, and polynucleotides encoding such proteins. Also provided are producer cells and compositions containing such targeted lipid particles and methods of making and using the targeted lipid particles.
KIR 7.1 GENE THERAPY VECTORS AND METHODS OF USING THE SAME
The present invention is directed to gene therapy constructs and pharmaceutical compositions for the expression of Kir7.1. The gene therapy constructs include a vector comprising a promoter operably connected to a polynucleotide encoding a Kir7.1 polypeptide. Methods of treating a subject having a condition associated with insufficient expression or function of a Kir7.1 polypeptide are also provided.
IL-10-CONTAINING VACCINES AND USES THEREOF
Provided herein are systems, compositions, and methods for expressing an antigen and a protein having interleukin-10-like activity in a cell. The systems, compositions, and methods described herein can be used to induce an immune response against an antigen and are useful, for example, in the prevention and treatment of a number of infectious diseases and cancers.
NIPAH VIRUS ENVELOPE PSEUDOTYPED LENTIVIRUSES AND METHODS OF THEIR USE
The present invention realtes 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 treatetment of cancer or CNS disorders.
COMPOSITIONS AND METHODS FOR TARGETING TUMOR ASSOCIATED TRANSCRIPTION FACTORS
Described are compositions and methods for targeting tumor associated transcription factors (e.g., PU.1) using IncRNA, constructs comprising IncRNA, and CRISPR/Cas systems, and polynucleotides encoding IncRNA, constructs comprising IncRNA, and CRISPR/Cas systems, vectors containing the polynucleotides, viral or non-viral delivery vehicles containing the vectors, and compositions (e.g., pharmaceutical compositions) containing the same for use in methods treatment.
OVEREXPRESSION OF LEMD2, LEMD3, OR CHMP7 AS A THERAPEUTIC MODALITY FOR TAUOPATHY
Provided herein are methods of inhibiting tau aggregation in a cell or a subject, comprising administering a LEM domain-containing protein 2 (LEMD2), a charged multivesicular body protein 7 (CHMP7), or an inner nuclear membrane protein Man 1 (LEMD3) or a nucleic acid encoding the LEMD2, the CHMP7, or the LEMD3 to the cell or the subject. Also provided herein are methods of treating or preventing a tauopathy in a subject, comprising administering LEMD2, CHMP7, or LEMD3 or a nucleic acid encoding the LEMD2, the CHMP7, or the LEMD3 to the subject, wherein the LEMD2, the CHMP7, or the LEMD3 inhibits tau aggregation in a cell in the subject. Also provided are nucleic acids encoding LEMD2, CHMP7, or LEMD3 (e.g., in an expression construct and operably linked to a heterologous promoter).
LENTIVIRAL VECTORS ENABLING ROUTING ANTIGENS TO MHC-II PATHWAY AND INDUCING CD4+ AND CD8+ T-CELL RESPONSES IN A HOST
The invention relates to a recombinant lentiviral vector genome comprising a polynucleotide encoding a fusion polypeptide, wherein said fusion protein comprises arranged from N-terminal to C-terminal ends: (i) a first polypeptide comprising a multimerization scaffold which comprises at least one collectin or a fragment thereof suitable to enable self-assembly of multimers of the first polypeptide, fused with at least one antigenic polypeptide; (ii) a second polypeptide comprising a CD40L ectodomain or a receptor binding fragment thereof, in particular the CD40L ectodomain of the human CD40L. The invention also relates to a lentiviral vector and pharmaceutical compositions comprising it.
METHODS TO RE-ENGAGE A FETAL WOUND HEALING PATHWAY FOR ADULT SKIN REPAIR
Compositions and methods are provided for promoting wound healing in a subject by administering a composition that enhances the expression of the protein nonselenocysteine-containing phospholipid hydroperoxide glutathione peroxidase (NPGPx) in wound-edge tissues. In accordance with one embodiment such compositions are used in conjunction with known treatments for use on chronic wounds including in diabetic patients.
VECTORS AND COMPOSITIONS FOR TREATING HEMOGLOBINOPATHIES
The invention provides improved gene therapy vectors, compositions, and methods.