Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

Abstract: The present disclosure is directed to antibodies binding to PD-L2 and methods of using such antibodies to treat cancers, such as those that express or overexpress PD-L2.

Abstract: The present disclosure provides a domain wall magnetic tunnel junction device. Integration of input spikes pushes a domain wall within a ferromagnetic track toward a magnetic tunnel junction (MTJ). An energy gradient within the track pushes the domain wall away from the MTJ by leaking accumulated energy from the input spikes. If the integrated input spikes exceed the energy leak of the gradient within a specified time period, the domain wall reaches the MTJ and reverses its resistance, producing an output spike. The leaking energy gradient can be created by a magnetic field, a trapezoidal shape of the ferromagnetic track, or nonuniform material properties in the ferromagnetic track. A feedback and feedforward crossbar configuration is disclosed that provides for a neuromorphic computing system suitable for unsupervised learning.

Abstract: Provided herein are compositions and methods for reducing expression of C9orf72 transcripts in cells containing expanded intronic GGGGCC regions, including those in subjects having or at risk of developing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Abstract: Provided herein are methods for the treatment of polycystic kidney disease, including autosomal dominant polycystic kidney disease, using modified oligonucleotides targeted to miR-17.

Abstract: Various embodiments disclosed relate to methods and compositions for antimicrobial treatment. In various embodiments, the present invention provides a method of antimicrobial treatment. The method includes at least one of exposing at least one microbe to a magnetic field, and contacting the at least one microbe with at least one nanoparticle including iron.

Abstract: A microelectromechanical (MEMS) device is provided. The MEMS device comprises a substrate and a movable structure flexurally connected to the substrate, capable of moving in relation to the substrate, wherein the movable structure further comprising two or more segments having at least one mechanical connection between said segments to provide structural integrity of the moving structure; and wherein the at least one mechanical connection electrically isolates at least two segments.

Abstract: In one aspect, the present disclosure provides for novel compositions of matter comprising multi domain peptide (MDP) hydrogels and cyclic dinucleotides (CDNs). Also disclosed are method of using such compositions in the treatment of cancer, including in particular the treatment of head and neck cancers, such as those resistant to CDN therapy.

Abstract: Provided herein are molecules having an antigen binding fragment that immunospecifically binds to BTN1A1, such as anti-BTN1A1 antibodies. These molecules include those having an antigen binding fragment that immunospecifically binds to glycosylated BTN1A1, such as anti-glycosylated BTN1A1 antibodies. Methods of making and using these molecules are also provided, including methods of using them in cancer therapies, or as cancer diagnostics.

Abstract: Described are compounds and methods useful for the treatment and investigation of Friedreich's Ataxia.

Abstract: Methods and devices for microfluidic detection of a biological maker in a biospecimen collected from a subject are disclosed. The microfluidic devices include nanoparticle-based nanosensors comprising supramolecular recognition sequences, protease consensus sequences, post-translationally modifiable sequences, or sterically hindered benzylether bonds for specific interaction with a biological marker. Also disclosed are particular nanosensors for detecting cytokines, and other proteins based upon supramolecular recognition without chemical modification or enzymatic cleavage.

Abstract: Described herein are novel lipid compositions comprising unsaturated dendrimers and methods of synthesis of unsaturated dendrimers. The lipid composition can comprise an ionizable cationic lipid, a phospholipid, and a selective organ targeting lipid. Also described herein are pharmaceutical formulations comprising an unsaturated dendrimer, a lipid composition, and a therapeutic agent. Further described in here are methods of mRNA delivery comprising a lipid composition and a therapeutic agent. Further described herein are high-potency dosage forms of a therapeutic formulated with a lipid composition.

Abstract: Disclosed herein are compounds and compositions useful in the treatment of GLS1 mediated diseases, such as cancer, having the structure of Formula I: Methods of inhibition GLS1 activity in a human or animal subject are also provided.

Abstract: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

Abstract: Systems, apparatuses, and methods provide various progressive, bit-level representations of digital data that are useful for a variety of systems and applications within the fields of machine learning, signal and data processing, and data analytics. Systems, apparatus, and methods for such representations incorporate one or more systems for machine learning, predicting, compressing and decompressing data, and are progressive such that the representations embody a sequential organization of information that prioritizes more information over less significant information. Embodiments of the present disclosure include systems for denoising, enhancing, compressing, decompressing, storing, and transmitting digitized media such as text, audio, image, and video. Methods can include partitioning data, modeling partitioned data, predicting partitioned data, transforming partitioned data, analyzing partitioned data, organizing partitioned data, and partially or fully restructuring the original data.

Abstract: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

Abstract: The present disclosure relates to polymers which contain a hydrophobic and hydrophilic segment which is sensitive to pH. In some aspects, the polymers form a micelle which is sensitive to pH and results in a change in fluorescence based upon the particular pH. In some aspects, the disclosure also provides methods of using the polymers for the imaging of cellular or extracellular environment or delivering a drug.

Abstract: This invention is in the field of medicinal pharmacology. In particular, the invention relates to a new class of small-molecules having a pyrazino-pyrimidine-dione (or related) structure which function as modulators (activators, inhibitors) of protease activated receptor type 2 (PAR2), and their use as therapeutics for the treatment of conditions involving PAR2 activity (e.g., asthma, chronic pain, cancer and/or vascular disorders).

Abstract: Provided is a method of making a preliposomal Annamycin lyophilizate, the composition made by the method, and the use of the composition made thereby in the treatment of cancer.

Abstract: The present disclosure provides ammonium compounds, e.g., compounds according to Formula I as set forth herein, which are useful as antimicrobial agents. Methods for the treatment of bacterial infections and associated conditions, e.g., gastrointestinal conditions, are also described, as well as methods for altering the microbiome of subjects such as humans.