Abstract: The invention includes compositions comprising at least one chimeric autoantibody receptor (CAAR) specific for an autoantibody, vectors comprising the same, compositions comprising CAAR vectors packaged in viral particles, and recombinant T cells comprising the CAAR. The invention also includes methods of making a genetically modified T cell expressing a CAAR (CAART) wherein the expressed CAAR comprises a desmoglein extracellular domain.

Abstract: The invention includes compositions comprising at least one chimeric autoantibody receptor (CAAR) specific for an autoantibody, vectors comprising the same, compositions comprising CAAR vectors packaged in viral particles, and recombinant T cells comprising the CAAR. The invention also includes methods of making a genetically modified T cell expressing a CAAR (CAART) wherein the expressed CAAR comprises a desmoglein extracellular domain.

Abstract: The present invention includes methods and compositions for treating cancer, whether a solid tumor or a hematologic malignancy. By expressing a chimeric antigen receptor in a monocyte, macrophage or dendritic cell, the modified cell is recruited to the tumor microenvironment where it acts as a potent immune effector by infiltrating the tumor and killing the target cells. One aspect includes a modified cell and pharmaceutical compositions comprising the modified cell for adoptive cell therapy and treating a disease or condition associated with immunosuppression.

Abstract: A cell bag rotator is constructed to slowly agitate a plurality of cell bags by rotation along a rotation axis that is at least partially horizontal. The cell bag rotator comprises a plurality of plates to which a respective cell bag is attached. The cell bag rotator is suitable for mixing cells with particles for performing various types of subsequent cell therapy processes on the cells.

Abstract: rAAV compositions useful for preventing the toxicity associated with organophosphate exposure are provided herein. The rAAV co-express a human butyrylcholinesterase and a proline-rich peptide. Each the esterase and the peptide may have an exogenous signal sequence. Also provided are compositions, e.g., aqueous liquid suspensions containing the rAAV.

Abstract: Emerging evidence indicates that diminished activity of the vasoprotective axis of the renin-angiotensin system, constituting angiotensin converting enzyme2 (ACE2) and its enzymatic product, angiotensin-(1-7) [Ang-(1-7)] contribute to pulmonary hypertension (PH). However, clinical success for long-term delivery of ACE2 or Ang-(1-7) would require stability and ease of administration to increase patient compliance. Chloroplast expression of therapeutic proteins enables their bioencapsulation within plant cells to protect from acids and gastric enzymes; fusion to a transmucosal carrier facilitates effective systemic absorption. Oral feeding of rats with bioencapsulated ACE2 or Ang-(1-7) attenuated monocrotaline (MCT)-induced increase in right ventricular systolic pressure, decreased pulmonary vessel wall thickness and improved right heart function in both prevention and reversal protocols.

Abstract: A method for delivering electrical stimulation to alter a cognitive state of a user, the method comprising: monitoring a brain signal from the user via one or more intracranial electrodes implanted in the brain of the user while the user is presented with a stimulus; comparing the brain signal to a testing phase biomarker, wherein the testing phase biomarker is derived from a cognitive test performed on a contributor during a testing phase; delivering electrical stimulation to a brain of the user based on the comparing step to steer the brain of the user towards a high performance cognitive state.

Abstract: The invention relates to proximity-based sortase-mediated protein purification and ligation. Specifically, the invention relates to techniques that links protein expression/purification with conjugation to therapeutic agents, imaging agents, or linkers.

Abstract: Sequences of a serotype 8 adeno-associated virus and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles.

Abstract: Disclosed herein are compounds and methods for modulating expression of disease-causing genes such as STXBP1, SCN1A, SCN2A, SCN8A, SLC6A1, and MECP2. Such compounds and methods are useful to treat, prevent, or ameliorate epileptic encephalopathy and neurodevelopmental disorders in an individual in need thereof.

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: Sequences of a serotype 8 adeno-associated virus and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles.

Abstract: Computer-implemented systems and methods for sleep optimization are disclosed. In certain embodiments, the system includes one or more peripheral devices, a network, one or more networked computers, and one or more remote servers. The peripheral devices and/or the networked computers collect one or more types of sleep data, calculate one or more sleep parameters, and transmit said sleep parameters to the one or more remote servers. The remote servers then perform calculations to determine optimal sleep recommendations. The sleep recommendations are then output to one or more networked computers and/or one or more peripheral devices as adjustments to sleep time and sleep duration.

Abstract: This invention relates to to conjugate antibody, drug and nanoparticle compositions and methods of generating the same. This invention further relates to methods of using same for imaging, diagnosing or treating a disease.

Abstract: Adeno-associated virus rh.20 sequences, vectors containing same, and methods of use are provided.

Abstract: The invention provides compositions and methods for adoptive T cell therapy in treating a variety of disorders including cancer, infections, and autoimmune disorders. In one embodiment, the invention provides a universal immune receptor that comprises a protein or peptide tag, such as a SpyCatcher or a SpyTag moiety, bound to an extracellular hinge region, a transmembrane domain, and an intracellular domain for T cell activation.

Abstract: The present invention includes compounds, compositions and methods that are useful for preventing or treating melanoma or any other cancer in a subject, such as a GPCR-expressing cancer. In certain embodiments, the compounds comprise estrogen (including estrogen derivatives or analogues), a selective GPER agonist and/or another G-protein coupled receptor (GPCR) agonist that increases cancer cell differentiation.

Abstract: This invention provides sets and libraries of short hairpin ribonucleic acid (shRNA) molecules comprising a double-stranded region of random sequence containing random mismatches, methods of generating same, sets and libraries of expression vectors for same, methods of generating same, and methods for identifying an RNA therapeutic or RNA molecule that has an ability to affect a biological parameter, for identifying a drug target for a disease or disorder of interest, and for identifying a variant of an RNA molecule that has an altered ability to affect a biological parameter of interest.

Abstract: Knee prostheses that more faithfully replicate the structure and function of the human knee joint in order to provide, among other benefits: greater flexion of the knee in a more natural way by promoting or accommodating internal tibial rotation, replication of the natural screw home mechanism, and controlled articulation of the tibia and femur respective to each other in a natural way. Such prostheses may include an insert component disposed between a femoral component and a tibial component, the insert component featuring, among other things, a reversely contoured posterolateral bearing surface that helps impart internal rotation to the tibia as the knee flexes. Other surfaces can be shaped using iterative automated techniques that allow testing and iterative design taking into account a manageable set of major forces acting on the knee during normal functioning, together with information that is known about natural knee joint kinetics and kinematics.

Abstract: Methods for reducing retinal inflammation and neuron death secondary to ocular hypertension, methods for reducing retinal inflammation and neuron death, reducing production of IL-1?, TNF-?, and C1q by CD11b+ CD11c+ and CD11b+ CD11c? cells during elevated intraocular pressure (IOP) and methods for decreasing transformation of astrocytes to an A1 neurotoxic phenotype (A1 astrocytes) and activation of the A1 astrocytes in a retina of a subject and decreasing production of complement component 3 (C3) by A1 astrocytes during elevated intraocular pressure in a subject in need thereof, comprising administering a glucagon-like peptide-1 receptor agonist.