Abstract: The present disclosure relates to compounds of Formula I, II, or III and compositions of the same, which are reversible off-on or ratiometric fluorescent iron sensors and are useful in live-cell imaging of labile iron ions and their quantification in subcellular compartments, as well as treatment of diseases associated with iron dyshomoestasis.

Abstract: In some aspects, the disclosure relates to compositions and methods of engineering a transgene. In some embodiments, the disclosure provides self-regulating recombinant nucleic acids, viral vectors and pharmaceutical compositions comprising a MeCP2 transgene. In some embodiments, compositions and methods described by the disclosure are useful for treating diseases and disorders associated with a loss of function mutation, for example Rett syndrome.

Abstract: Aspects of the disclosure relate to manufacturing AAV using a stable cell line that expresses the rep gene by engineering tRNA to suppress a mutation introduced to a rep integrated into the genome of the cell line.

Abstract: In some aspects, the disclosure relates to compositions and methods for treating fibrodysplasia ossificans progressiva (FOP) in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that inhibit (e.g., decrease) expression of mutated AVCR1 gene in muscle cells or connective tissues.

Abstract: Droplet-interface bilayer and lipid bilayer membrane compositions stabilized with an amphiphilic polymer are disclosed. Methods of making and using the compositions are also disclosed.

Abstract: The invention provides polymers and polymer-based nano-structures, in particular, polymers and polymer network to which biomolecules (e.g., proteins, antibodies, peptide aptamers) can be covalently conjugated and stably encapsulated therein and be controllably delivered and released upon degradation of the nano-structures in response to specific microenvironment, and compositions and methods of preparation and use thereof.

Abstract: Methods for using cyclin-dependent kinase (CDK) inhibitors to enhance growth and self-renewal of progenitor cells, in vitro and in vivo.

Abstract: The invention is directed to a system and methods for treating neurovascular compression. Certain preferred embodiments of the invention generally comprise an apparatus having an elongated body portion including a proximal anchor element and distal anchor element. The apparatus also may include one or more marker elements to facilitate placement of the apparatus by the user. The apparatus may be configured to permit the user, after insertion and deployment of the apparatus in a blood vessel, to move the blood vessel so that the blood vessel is no longer in contact with and compressing a nerve or other parts of the nervous system.

Abstract: The disclosure, in some aspects, relates to nucleic acids, compositions and kits useful for gene therapy with reduced immune response to transgene products.

Abstract: In some aspects the disclosure provides compositions and methods for promoting expression of functional Forkhead box G1 (FOXG1) protein in a subject. In some embodiments, the disclosure provides methods of treating a subject having FOXG1 deficiency.

Abstract: Aspects relate to patterned nanostructures having a feature size not including film thickness of below 5 microns. The patterned nanostructures are made up of nanoparticles having an average particle size of less than 100 nm. A nanoparticle composition, which, in some cases, includes a binder, is applied to a substrate. A patterned mold used in concert with electromagnetic radiation function to manipulate the nanoparticle composition in forming the patterned nanostructure. In some embodiments, the patterned mold nanoimprints a pattern onto the nanoparticle composition and the composition is cured through UV or thermal energy. Three-dimensional patterned nanostructures may be formed. A number of patterned nanostructure layers may be prepared and joined together. In some cases, a patterned nanostructure may be formed as a layer that is releasable from the substrate upon which it is initially formed.

Abstract: Aspects of the disclosure relate to compositions and methods for modulating expression of certain RPGR retinitis pigmentosa GTPase regulator (RPGR) proteins in a cell or subject. In some embodiments the disclosure provides expression vectors encoding one or more RPGR proteins and/or one or more inhibitory nucleic acids that target certain isoforms of RPGR, for example RPGRCONST. In some aspects, the disclosure relates to methods of treating retinitis pigmentosa (RP) in a subject by administering compositions that modulate RPGR expression or activity.

Abstract: Aspects of the disclosure relate to compositions and methods for delivering a transgene (e.g., a transgene encoding one or more gene products) to a target cell. The disclosure is based, in part, on adeno-associated virus (AAV) capsid protein variants characterized by tropisms for certain cell types (e.g., neurons, muscle cells, bone cells, heart cells, etc.). In some embodiments, recombinant A A Vs (rAAVs) comprising the capsid protein variants (e.g., AAVv66, SEQ ID NO: 1) are more efficiently packaged than rAAVs having certain wild-type AAV capsid proteins. Methods of delivering an rAAV comprising the AAV capsid protein variants are also described by the disclosure.

Abstract: A method for use in traversing a vehicle transportation network by an autonomous vehicle (AV) includes traversing, by the AV, the vehicle transportation network. Traversing the vehicle transportation network includes identifying a distinct vehicle operational scenario; instantiating a first decision component instance; receiving a first set of candidate vehicle control actions from the first decision component instance; selecting an action; and controlling the AV to traverse a portion of the vehicle transportation network based on the action. The first decision component instance is an instance of a first decision component modeling the distinct vehicle operational scenario.

Abstract: An optical device as described herein includes a host substrate fabricated from a dielectric material transparent in the Infrared range. Additionally, the optical device as discussed herein includes multiple elements disposed on the host substrate. The multiple elements are spaced apart from each other on the host substrate in accordance with a desired pattern. Each of the multiple elements disposed in the host substrate is fabricated from a second material having a refractive index of greater than 4.5. Such an optical device provides an improvement over conventional optical devices that operate in the Infrared range.

Abstract: Aspects of the disclosure relate to compositions and methods for exon replacement in a cell or a subject. In some embodiments, the disclosure relates to isolated nucleic acids (and vectors, such as rAAV vectors) encoding one or more guideRNAs (gRNAs) that target an intron-exon boundary; an intronic sequence having a splice signal; and a donor sequence encoding a gene product of a gene of interest, or portion thereof. In some embodiments, compositions described herein are useful for replacing mutant exons associated with certain diseases, for example Duchen's muscular dystrophy (DMD), cystic fibrosis (CF), spinal muscular atrophy (SMA), Rett syndrome, and mucopolysaccharidosis (MPS).

Abstract: Instruments and methods for wide-field polarized imaging of the skin to determine an outer lesion margin objectively in vivo to provide guidance to a surgeon. Quantitative characterization of collagen structures in the skin can be used to determine the outer lesion margin or monitor skin treatment.

Abstract: A memristive device includes a biomaterial comprising protein nanowires and at least two electrodes in operative arrangement with the biomaterial such that an applied voltage induces conductance switching. An artificial neuron or an artificial synapse includes a memrisitive device with the electrodes configured to apply a pulsed voltage configured to mimic an action-potential input.

Abstract: The present disclosure provides antisense compounds, methods, and compositions for silencing C9ORF72 transcripts. The present disclosure provides antisense compounds, methods, and compositions for the treatment, prevention, or amelioration of diseases, disorders, and conditions associated with C9ORF72 in a subject in need thereof. Also contemplated are antisense compounds and methods for the preparation of a medicament for the treatment, prevention, or amelioration of a disease, disorder, or condition associated with C9ORF72.

Abstract: To overcome the problem of a diffractive surface having a large, and often excessively large, amount of chromatic aberration, an optical system can use multiple cascaded or sequential diffractive surfaces that, combined, have a reduced amount of chromatic aberration. The optical system can be designed such that all rays traversing the optical system and passing through the diffractive surfaces have an equal optical path length. In the design process, the sets of rays are identified, and the designs of the diffractive surfaces are selected to produce the angular deviations to produce the identified ray paths. In one example, an achromatic lens formed as two annular optical surfaces can receive a collimated incident beam, redirect rays helically at the first surface toward the second surface, and redirect the rays at the second surface toward a focal point. The azimuthal redirection can decrease with increasing distance away from a central axis.