Abstract: Methods of treating GLA-deficiency (Fabry disease) in a subject in need thereof comprising administration of a recombinant adeno-associated virus (rAAV) vector having an AAV capsid and a vector genome comprising a sequence encoding a functional human alpha-galactosidase A (hGLA) are provided. Also provided are pharmaceutical compositions containing an rAAV vector and use of these pharmaceutical compositions for treating Fabry disease.

Abstract: The present disclosure relates, in part, to immune cell targeted lipid nanoparticle (LNP) compositions, and methods of use thereof for in vivo delivery of nucleic acid molecules and/or therapeutic agents to a target cell. In certain embodiments, the nucleic acid molecules encode chimeric antigen receptors (CARs). In certain embodiments, the target cell is a T cell. In certain embodiments, the present disclosure relates to the use of the LNPs described herein for the treatment, prevention, and/or amelioration of diseases and/or disorders, including but not limited to cancer.

Abstract: A two-step chromatography purification scheme is described which selectively captures and isolates the genome-containing rAAV vector particles from the clarified, concentrated supernatant of a rAAV production cell culture. The process utilizes an affinity capture method performed at a high salt concentration followed by an anion exchange resin method performed at high pH to provide rAAV vector particles which are substantially free of rAAV intermediates.

Abstract: Compositions and methods are disclosed for treating cancer patients and identifying patients with a malignancy that are likely to respond to treatment with anti-PD-1/PD-L1 and other anti-cancer and immune-modulating therapeutics.

Abstract: The invention provides T cells comprising nucleic acid sequence encoding a chimeric antigen receptor and a nucleic acid sequence encoding an enhancer of T cell priming, compositions including the T cells, and methods of using the T cells to treat diseases associated with the expression of disease-associated antigens.

Abstract: The present invention relates to compositions and methods for inducing an adaptive immune response against Zika virus (ZIKV) in a subject. In certain embodiments, the present invention provides a composition comprising a nucleoside-modified nucleic acid molecule encoding a ZIKV antigen, adjuvant, or a combination thereof. For example, in certain embodiments, the composition comprises a vaccine comprising a nucleoside-modified nucleic acid molecule encoding a ZIKV antigen, adjuvant, or a combination thereof.

Abstract: The present invention provides compositions and methods for transducing cells (e.g. T cells or immune cells). Also provided herein are methods of treating a disease in a subject in need thereof.

Abstract: Provided herein are rAAV and other vectors and compositions useful for treating a patient having CMT2 comprising: (a) a recombinant nucleic acid sequence encoding an engineered human mitofusin 2 coding sequence operably linked to regulatory sequences which direct expression thereof in a human target cell. Also provided are rAAV and other vectors and compositions useful for treating a patient having CMT2 comprising: (b) a nucleic acid sequence encoding at least one miRNA specific for an endogenous human mitofusin 2 sequence in a human CMT2A subject, wherein the miRNA coding sequence is operably linked to regulatory sequences which direct expression thereof in the subject. Further provided are compositions containing both the engineered hMfn2 coding sequence and the at least one miRNA coding sequence, wherein the engineered human mitofusin 2 coding sequence has a sequence which differs from endogenous human mitofusin 2 in the CMT2A patient in the target site of the encoded miRNA.

Abstract: In one example, a bearing or transmission has an outer race that extends circumferentially about a central axis. An inner race is disposed within the outer race and extends circumferentially about the central axis so as to be coaxial with the outer race. A plurality of load-bearing rolling elements are disposed between the inner race and the outer race, and are spaced circumferentially from one another so as to not contact one another. A plurality of separator rolling elements are disposed within the outer race. Each separator rolling element is disposed between a respective pair of the load-bearing rolling elements such that the separator rolling element is in rolling contact with the load-bearing rolling elements of the respective pair. The load-bearing and separator rolling elements form a contiguous ring.

Abstract: Provided herein are novel AAV capsids and recombinant AAV vectors comprising the same. In one embodiment, vectors employing a novel AAV capsid show increased transduction of a selected target tissue as compared to a prior art AAV.

Abstract: A laser is used to controllably generate and propagate cleaves to produce a cleaved surface along the {111} crystallographic plane in diamonds.

Abstract: Provided are systems and methods for inducing strain fields to give rise to controllable wrinkle patterns in a variety of substrates. Also provided are articles having persistent wrinkling patterns thereon.

Abstract: The present disclosure is concerned with magneto-patterned cell-laden hydrogel materials and methods of making and using those materials. The disclosed materials are useful for, among other things, repair of tissue defects, e.g., tissue at a tissue interface such as a bone-cartilage interface.

Abstract: The present invention relates to antibodies, binding polypeptides, and scFvs specific for fibroblast activation protein (FAP) capable of cross reacting with canine, mouse, and human FAP.

Abstract: The present invention relates to compositions and methods for inducing an adaptive immune response in a subject. In certain embodiments, the present invention provides a composition comprising a nucleoside-modified nucleic acid molecule encoding an antigen, adjuvant, or a combination thereof. For example, in certain embodiments, the composition comprises a vaccine comprising a nucleoside-modified nucleic acid molecule encoding an antigen, adjuvant, or a combination thereof.

Abstract: Described herein is a beads-free bioprocessor as an automated and cost-effective T cell processing and manufacturing platform. T cells are a core component in CAR T cell therapies for cancer treatment, but are difficult to manufacture to scale in clinically relevant quantities. The 3D bioprocessor provides an alternative device that is scalable, beads-free, easy-to-use, and cost-effective for using CAR T cell therapy in cancer immunotherapy. Besides CAR T cell application, this platform technology has potential for many other applications such as cancer cell isolation.

Abstract: The present disclosure is generally directed to quinolinyl and pyridinyl containing compounds targeting on alpha-synuclein for neurological diseases. These compounds, particularly radiolabeled versions of these compounds, can be particularly useful in PET scans targeting diseases such as Parkinson's disease (PD), Dementia with Lewy Bodies (DLB) and Multiple System Atrophy (MSA).

Abstract: The invention provides compositions and methods for treating diseases associated with expression of a cancer associated antigen as described herein. The invention also relates to chimeric antigen receptor (CAR) specific to a cancer associated antigen as described herein, vectors encoding the same, and recombinant T cells comprising the CARs of the present invention. The invention also includes methods of administering a genetically modified T cell expressing a CAR that comprises an antigen binding domain that binds to a cancer associated antigen as described herein.

Abstract: The present disclosure provides microRNA-based therapies using a hydrogel delivery system that provides regenerative approach to myocardial infarction by targeting cardiomyocytes. The hydrogel provides for local and sustained cardiac delivery of microRNAs, such miR-302 mimics that can be used to promote cardiomyocyte proliferation. Also provided are compositions suitable for local and sustained release and methods for intramyocardial gel delivery of a miRNA oligonucleotide.

Abstract: A synthetic or recombinant human lecithin cholesterol acyltransferase (LCAT) variant is provided which comprises an LCAT enzyme having a substitution at position 114 based on the residue numbering of wild-type (WT) human LCAT [SEQ ID NO:1], wherein said variant is characterized by one or more of: (i) an esterification rate higher than the esterification rate of WT human LCAT; and/or (ii) an association with higher density lipoprotein levels as compared to subjects having WT LCAT. Also provided are vectors encoding the variant, compositions containing same, and methods of using the variant proteins and vectors for treatment of a variety of disorders associated with defective wt LCAT.