Abstract: Compositions and methods for treating metabolic diseases in a subject are provided. A viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding a GLP-1 receptor agonist fusion protein and regulatory sequences which direct expression thereof.

Abstract: A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDS gene and two or more immunosuppressants is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating Hunter syndrome and the symptoms associated with Hunter syndrome.

Abstract: Methods for predicting risk of heart transplant rejection are disclosed.

Abstract: The presently disclosed subject matter provides a biomimetic lung disease model, and methods of its production and use. In one exemplary embodiment, the biomimetic lung disease model can include a first and second microchannel with a membrane coated with airway epithelial cells disposed between the microchannels and at least one device coupled to the biomimetic model that delivers an agent to at least one microchannel. In certain embodiments, the agent is cigarette smoke.

Abstract: De novo artificial protein based reporters that may be expressed in eukaryotic (e.g., mammalian) cells and methods of using the same are provided herein.

Abstract: The invention relates to compositions and methods for diagnosing as well as treating cancer diseases associated with choline kinase (ChoK). Specifically, the invention relates to a composition comprising an intrinsically fluorescent choline kinase (ChoK) inhibitor or a ChoK inhibitor operably linked to a fluorescent dye. The composition is capable of diagnosing and/or treating cancer diseases associated with ChoK.

Abstract: A recombinant vector comprises simian adenovirus 43, 45, 46, 47, 48, 49 or 50 sequences and a heterologous gene under the control of regulatory sequences. A cell line which expresses simian adenovirus 43, 45, 46, 47, 48, 49 or 50 gene(s) is also disclosed. Methods of using the vectors and cell lines are provided.

Abstract: The present disclosure provides a device for imaging cardiac structures having at least a sheath, a wire-basket structure and an optical scope. The present disclosure also provides methods for imaging of epicardial space, epicardial ablation and cardiac ultrasound using the device disclosed herein.

Abstract: Disclosed herein is a vaccine comprising a Middle East Respiratory Syndrome coronavirus (MERS-CoV) antigen. The antigen can be a consensus antigen. The consensus antigen can be a consensus spike antigen. Also disclosed herein is a method of treating a subject in need thereof, by administering the vaccine to the subject.

Abstract: The present invention relates to compositions and methods for treating diseases, disorders or conditions associated with the expression of the glycosyl-phosphatidylinositol (GPI)-linked GDNF family protein ?-receptor 4 (GFR?4).

Abstract: The present invention relates to Charcot-Marie-Tooth disease 2A (CMT2A) harboring the p.Arg364Trp or p.His361Tyr Mfn2 mutation, whose human counterpart results in severe, early-onset axonal neuropathy for p.Arg364Trp Mfn2 mutation in fertilized rat eggs. Cohorts of mutants and wild type littermates were characterized with multiple motor deficits that worsened over time. Separate cohorts of mutant and wild type at 7, 40, and 48 weeks showed reduced density of myelinated axons and active axonal degeneration in distal but not proximal nerves, as well as axonal degeneration in the fasciculus gracilis of the cervical spinal cord at 40 and 48 weeks not present in 7-week-old cohort Mfn2 mutants, or wild type at 7 or 40 weeks. The p.His361Tyr Mfn2 mutation using CRISPR/Cas9 showed abnormalities in gait dynamics at 8 weeks and lengthening of gait cycle at 16 weeks. The invention provides progressive axonal neuropathy for examining pathogenesis and treatment of CMT2A.

Abstract: This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of RNA, oligoribonucleotide, and polyribonucleotide molecules.

Abstract: Provided herein are methods, systems and apparatus for diagnosing, predicting and/or preventing acute allograft vasculopathy in an organ transplant patient. The method comprises executing on a processor the steps comprising: analyzing clinical risk factors of a cardiac, kidney or liver transplant patient in a model which distinguishes between clinical biomarkers of allograft vasculopathy patients and non-allograft vasculopathy patients, and analyzing digital biopsy images from the transplant patient to morphologically differentiate patients which are prE allograft vasculopathy or which have active allograft vasculopathy from patients without acute allograft vasculopathy; assigning prE-, active or non-allograft vasculopathy status to a patient based on the outcome of the analysis of the clinical biomarkers and morphologic biomarkers; and where preE or active statis is assigned, treating the patient.

Abstract: The present application discloses compositions and methods for use of nucleoside modified mRNA that encode for at least one liver regenerative factor. The present invention also relates to compositions and methods for use of nucleoside modified mRNA complexed to nanoparticles. The disclosed compositions and methods are useful for treating acute liver diseases, chronic liver diseases, and/or acetaminophen (acetyl-para-aminophenol, APAP) overdose.

Abstract: A composition, including a substrate having a planar array of depressions each defined by concave walls and a moat disposed around each depression of said array of depressions.

Abstract: A method for creating an irradiation plan. The method may include receiving an irradiation therapy plan for irradiating a target with a proton beam from a gantry. The irradiation therapy plan may have a plurality of irradiation spots for delivering protons to the target. The method may include identifying a rotational profile for angular rotation of the gantry with respect to the target, a slew time for a proton beam to travel between each of the plurality of irradiation spots, and an amount of protons to be delivered to each irradiation spot. The method may also include creating an irradiation plan. The irradiation plan may include a plurality of groups of irradiation spots based on the rotational profile, the slew time, and the amount of protons to be delivered to each irradiation spot. Each group of irradiation spots may be associated with a respective portion of the angular rotation.

Abstract: Compositions for the prevention and treatment of genital herpes, comprising nucleoside modified mRNAs that encode herpes simplex virus (HSV) glycoproteins, including those involved in virus entry and immune evasion, and methods of use thereof.

Abstract: A microfluidic device contains a first layer having a plurality of channels, a second layer having a plurality of droplet makers, and a third layer having a plurality of through-holes connecting the plurality of channels to the plurality of droplet makers. The channels have a height of at least 4 times greater than the height of the droplet makers. The microfluidic device has at least 500 droplet makers in an area less than 10 cm2. The channels are formed by direct laser-micromachining and the droplet makers are formed by soft lithography molding.

Abstract: The present invention provides compositions and methods for treating cancer in a human. The invention includes administering a T cell, genetically modified to express a chimeric antigen receptor (CAR), a bispecific antibody, or a combination thereof to a subject. The CAR and bispecific antibody of the invention can comprise a human antibody, a humanized antibody, or antigen-binding fragments thereof.

Abstract: Methods and compositions are provided for treatment of peroxisomal biogenesis disorders (PBDs). More particularly, recombinant adeno-associated viruses (rAAV) provided in the form of compositions are used to deliver a nucleic acid encoding human PEX1 to host cells. The rAAVs comprise a AAV capsid, and packaged therein a vector genome comprising an AAV 5? inverted terminal repeat (ITR) sequence; a promoter; a coding sequence encoding a human PEX1; and an AAV 3? ITR.