Methods for alleviating symptoms in a Fragile X Syndrome (FXS) patient using adeno-associated viral (AAV) 9 viral particles encoding a wild-type human fragile X mental retardation 1 (FMR1) protein (human FMRP). Also provided herein are methods to determine suitable doses of AAV9 viral particles for a FXS patient to alleviate at least one symptom associated with FXS, as well as methods for monitoring treatment efficacy.

The invention relates to compositions and methods for exosome preservation by freeze-drying as well as methods of use of the freeze-dried exosome compositions.

Disclosed herein are compositions of rAAV particles and methods for administrating rAAV particles having enhanced transduction properties.

Described herein are compositions and techniques related to generation and therapeutic application of artificial synapses. Artificial synapses are engineered extracellular vesicles, including exosomes, which incorporate sticky binders on their surface to anchor signaling domains against biological targets, such as receptors. These engineered additives can be organized in genetic vector constructs, expressed in mammalian cells, wherein the sticky binders attach to extracellular vesicles such as exosomes, thereby presenting their joined signaling domains which are rapidly taken up by recipient cells. Artificial synapses adopt the hallmark biophysical and biochemical features of extracellular vesicles, allowing for rapid deployment and scale-up. Importantly, this strategy can allow for kinetically favorable signal generation and signal propagation. This includes, for example, increasing density of agonist presentation to support receptor clustering an onerous barrier for traditional receptor targeting strategies.

Disclosed, at least in part, are dosings of viral vectors concomitantly with synthetic nanocarriers attached to an immunosuppressant, in combination with dosings of the synthetic nanocarriers attached to an immunosuppressant without a viral vector or further dosings of the synthetic nanocarriers attached to an immunosuppressant concomitantly with doses of the viral vector, and related compositions that provide reduced humoral immune responses and/or increased or durable transgene or nucleic acid material expression.

Disclosed herein are compositions and methods with enhanced capability to reduce uric acid concentration. The compositions are loaded in a nanocarrier for oral drug delivery. The composition comprises a first vector and a second vector. The first vector encodes one or more mRNAs. The one or more mRNAs encode a peptide with uricase activity and are labeled with one or more capsid protein tags. The second vector encodes one or more capsid proteins. The one or more capsid proteins bind to the one or more capsid protein tags on the one or more mRNA.

Disclosed are bruceolides for the treatment of cancer, and other diseases, selectively targeting unwanted cells. The disclosed bruceolides may include a site specific cleavable moiety inhibiting the chemotoxic activity until cleaved, i.e., removed, within and/or near a cancer to be treated. As to facilitate selective delivery to cancer tumors, the disclosed bruceolides may be loaded into, attached to or otherwise carried by nanoparticles.

The invention relates to a method for providing a virus-like particle (VLP) derived from John Cunningham virus (JCV), relates to a VLP associated with a cargo, in particular a protein, and a drug delivery system (DDS) obtainable by said method, in particular for crossing the blood brain barrier (BBB), and a VLP containing composition. The method comprises steps of disassembly of VLP into pentamers, inducing the pentamers to aggregate and reassembly into VLP.

According to some embodiments, a carrier for reducing a likelihood of a pathogen binding to cell structures of a host comprises a core, surface features extending from an exterior surface of the core, wherein the surface features are configured to bind to target areas of cell structures of the host to at least partially block the pathogen from binding to said target areas as a result of competitive inhibition, and a plurality of binding sites along the exterior surface, wherein the binding sites are configured to attract at least one portion of the pathogen, wherein the binding sites are recognizable by the pathogen and are able to be bound by the pathogen, thereby at least partially immobilizing the pathogen and reducing the likelihood of the pathogen binding to target areas of cell structures of the host.

Disclosed is a nanoparticle comprising an inner core comprising a virus; and an outer surface comprising a cellular membrane derived from a cell, and process of making thereof. The virus is an oncolytic virus and cellular membrane is derived from for example red blood cells.