Abstract: Aspects of the invention described herein relate to methods of making and using inducible promoters for transgene expression. The inducible promoters are derived from the NFAT-RE inducible system and are used to improve or enhance T cell survival and proliferation.

Abstract: Some embodiments of the invention include pseudotyped particles (e.g., pseudo typed exosomes, pseudotyped VSV, and pseudo typed lentiviruses) and modified cells. Other embodiments of the invention include compositions (e.g., pharmaceutical compositions) of pseudotyped particles (e.g., pseudotyped exosomes, pseudotyped VSV, and pseudotyped lentiviruses) and modified cells. Certain embodiments of the invention include methods of making pseudotyped particles (e.g., pseudotyped exosomes, pseudotyped VSV, and pseudotyped lentiviruses) and modified cells. Other embodiments of the invention include methods of administering pseudotyped particles (e.g., pseudotyped exosomes, pseudotyped VSV, and pseudotyped lentiviruses). Further embodiments of the invention include methods of administering pseudotyped particles (e.g., pseudotyped exosomes, pseudotyped VSV, and pseudotyped lentiviruses) to treat diseases (e.g., muscular dystrophy). Additional embodiments of the invention are also discussed herein.

Abstract: Provided are methods for one or more of the following: preventing, inhibiting, disrupting, dispersing or treating a polymicrobial biofilm comprising a Haemophilus bacteria in vitro and/or in vivo, such as in a subject in need thereof. The method comprises or consists essentially of, or yet further consists of contacting the polymicrobial biofilm with: (i) an anti DNA binding and bending protein (DNABII) antibody or a biologically active fragment thereof; and (ii) an anti majority subunit (PilA) of type IV pilus (T4P) antibody or a biologically active fragment thereof. Additionally, the methods may further comprise contacting the polymicrobial biofilm with an antibiotic optionally comprising a ?-lactam antibiotic or a sulfonamide antibiotic. Also provided are compositions and kits suitable for use in the methods.

Abstract: Compositions and methods for the detection and treatment of ADHD are provided.

Abstract: A patient table adapted for use in association with an MR scanner for neonatal infants is provided. The patient table has an extendable patient bed attached to and extendable from the patient table. The patient bed may be at least partially inserted into an MR scanner without requiring the patient table to enter the MR scanner. A transport mechanism is on the underside of the patient table so that it may be readily moved over the floor on which it rests. The patient table includes a latching mechanism that may operate to releasably attach the patient table to a patient table docking assembly. The docking assembly is operative to selectively move the patient table towards, into and away from the MR scanner.

Abstract: Some embodiments of the invention include a nucleic acid molecule comprising natural nucleotides, non-natural nucleotides, an LNA which comprises one or more RNA core molecules, or an RNA molecule which comprises more than one RNA core molecule. Some embodiments of the invention include a nucleic acid molecule comprising an RNA molecule which comprises more than one RNA core molecule. Other embodiments of the invention include a nucleic acid molecule comprising a DNA molecule encoding the RNA molecule (e.g., vector or viral vector). Other embodiments include compositions or pharmaceutical compositions that comprise the nucleic acid molecule. Some embodiments of the invention comprise reducing miR-143 in a cell. Other embodiments of the invention include methods to deliver a protein across the BBB. Other embodiments include methods for treating disease (e.g., LSD, neuronopathic disease, neurodegenerative disease, Hurler syndrome, or MPS I). Additional embodiments of the invention are also discussed herein.

Abstract: A liposomal composition (“ADx-003”) is provided, ADx-003 comprising a first phospholipid; a sterically bulky excipient that is capable of stabilizing the liposomal composition; a second phospholipid that is derivatized with a first polymer; a macrocyclic gadolinium-based imaging agent; and a third phospholipid that is derivatized with a second polymer, the second polymer being conjugated to a targeting ligand, the targeting ligand being represented by Formula I: wherein X is —CH2—, —CH2—CH2—, —CHO—, or —O—CO—; Y is —CH—CH?CH— or A and B are independently selected from C and N; R1, R2, R3, and R4 are independently selected from —H, halogen, —OH, and —CH3; and R5, R6, and R7 are independently selected from —H, halogen, —OH, —OCH3, —NO2, —N(CH3)2, C1-C6 alkyl, or a substituted or unsubstituted C4-C6 aryl group, except that when A and/or B is N the adjacent R5 and/or R7 is —H, or a pharmaceutically acceptable salt thereof.

Abstract: A glomerulus on a chip (GOAC) to recapitulate the human glomerular filtration barrier, the structure responsible for filtering the blood and preventing the loss of proteins, is provided using human podocytes and glomerular endothelial cells seeded into microfluidic chips. In long-term cultures, cells maintain their morphology, form capillary-like structures and express slit diaphragm proteins. This system recapitulates functions and structure of the glomerulus, including permselectivity. When exposed to sera from patients with anti-podocyte autoantibodies, the chips show albuminuria proportional to patients' proteinuria, phenomenon not observed with sera from healthy controls or individuals with primary podocyte defects. Also shown is its applicability for renal disease modeling and drug testing.

Abstract: Described are methods and systems for the treatment of individuals having a disorder characterized by complement system dysregulation. The described methods and systems may be used for a variety of purposes, including for example, establishing one or both of a general or personalized dosing schedule for treatment using a complement inhibitor, establishing a dosage schedule sufficient to maintain an effective amount of complement inhibitor, establishing general dosing schedules for novel complement modifying agents and identifying a treatment regimen and/or dose eliminating the possibility of under dosing medication, and treatment regimen and/or dose for reducing or preventing toxicity in a patient.

Abstract: Disclosed are methods of treating pain in a mammal, which may include the step of administering human growth hormone to a mammal in need thereof The pain treated by the disclosed methods may be of a type caused by inflammation induced mechanical and/or thermal hypersensitivity, and may include, for example, a pain type resulting from one or more conditions selected from peripheral injury pain, post-operative pain, cutaneous inflammation, cutaneous incision, muscle incision, or chronic pain. Disease states in which the disclosed methods may be used include fibromyalgia, sickle cell anemia, epidermolysis bullosa, erythromelalgia, complex regional pain syndrome, or generalized muscle pain.

Abstract: The disclosure provides immunogenic peptides comprising at least a portion of a Plasmodium HAP2 paralog (“HAP2p”) protein, immunogenic compositions comprising or encoding the immunogenic peptides, antibodies binding the immunogenic peptides, and methods of preventing Plasmodium transmission incorporating the peptides, compositions, and/or antibodies. In some embodiments, the immunogenic peptide has a sequence comprising a sequence with at least 80% identity to a sequence of at least 10 continuous amino acids of SEQ ID NO:2, a Plasmodium HAP2 paralog (“HAP2p”) protein.

Abstract: Variant factor H binding proteins that can elicit antibodies that are bactericidal for at least one strain of Neisseria meningitidis, compositions comprising such proteins, and methods of use of such proteins, are provided.

Abstract: A method for motion correction of Magnetic Resonance (MR) images is provided. The method includes acquiring a k-space dataset for an object using an MR scanner, detecting or identifying corrupted k-space data from the acquired k-space dataset, extracting the corrupted k-space data from the acquired k-space dataset, recovering the corrupted k-space data, combining uncorrupted k-space data of the acquired k-space dataset with the recovered k-space data to form a full k-space dataset, and reconstructing an image for the object based on the full k-space dataset. A magnetic resonance imaging system for correcting corrupted k-space data of an entire k-space dataset is also provided.

Abstract: The invention relates to compositions and methods as part of a pharmacotherapeutic strategy that targets the endochondral ossification process in a pharmacologically selective and site-specific manner. A variety of orthopedic pathologies are caused by or associated with generalized or local dysregulation of endochondral ossification, for example trauma to the bone growth plate or diaphysis can cause a serious imbalance in bone growth, leading to progressive deformity that today can only be treated surgically. Dysregulated endochondral ossification is also behind heterotopic ossification, which arises in soft tissues and causes pain, decrease in mobility and other clinical problems. The invention therefore provides RARy agonist and antagonist nanoparticle compositions for treating abnormal endochondral ossification and bone growth that can deliver robust local therapeutic control over a particular long bone's growth with a long-lasting effect.

Abstract: Disclosed herein are vaccine compositions, in particular, polyvalent icosahedral compositions for antigen presentation. The disclosed compositions may contain an S particle made up of recombinant fusion proteins. The recombinant fusion proteins may include a norovirus (NoV) S domain protein, a linker protein domain operatively connected to the norovirus S domain protein, and an antigen protein domain operatively connected to said linker.

Abstract: Disclosed are methods of making a genetically modified immune cell for modifying a tumor microenvironment (TME) and methods of modifying a tumor microenvironment (TME). In some embodiments, the method can include delivering a first vector to an immune cell, wherein the first vector comprises a nucleic acid encoding a protein that induces T-cell proliferation, promotes persistence and activation of endogenous or adoptively transferred NK or T cells and/or induces production of an interleukin, an interferon, a PD-1 checkpoint binding protein, HMGB1, MyD88, a cytokine or a chemokine. Methods of modulating the suppression of the immune response in a tumor microenvironment, minimizing the proliferation of tumor and suppressive cells, and increasing the efficiency of an anti-cancer therapy, anti-infection therapy, antibacterial therapy, anti-viral therapy, or anti-tumoral therapy are also provided.

Abstract: The present invention relates to methods for identifying an EoE endotype of a patient and treating the patient with one or more therapies targeted to the patient's disease endotype; and related methods for stratifying patients for clinical trials.

Abstract: The invention provides for AAV vectors expressing the ANO5 gene and antioxidant therapy as methods of inducing muscle regeneration and a method of treating muscular dystrophy.

Abstract: The present disclosure is directed to antibodies binding to Glypican 2 and methods of using such antibodies to treat cancers that express or overexpress the Glypican 2 antigen.

Abstract: Compositions and methods for the treatment and diagnosis of eosinophilic esophagitis are disclosed.