Abstract: Disclosed herein are nucleic acid compositions and methods of use. The nucleic acid compositions may have a therapeutic nucleic acid sequence operably linked to a nuclear targeting sequence that increases expression of the therapeutic nucleic acid in a cell by at least 1.25 fold; at least 80% sequence identity to SEQ ID NO: 6; or a first regulatory element comprising a promoter sequence operably linked to a hemoglobin subunit gamma intron (hBGi) sequence, and a second regulatory element comprising a woodchuck hepatitis posttranscriptional regulatory element (WPRE) sequence. The nucleic acid compositions with these features may enhance therapeutic nucleic acid transfection and expression. Also disclosed herein are transgenes optimized for gene therapy applications, including novel FVIII transgene sequences, in which the B domain may be non-naturally occurring the A1 and/or A3 domain may include at least one amino acid substitution.

Abstract: Provided are methods and compositions for expressing a functional dystrophin protein or a functional fragment thereof in a subject. The method can include administering a nucleic acid encoding the dystrophin protein to the subject, administering a sonoactive agent to the subject, and applying ultrasound energy the cell. Also provided are use of a nucleic acid encoding a functional dystrophin protein and a sonoactive agent in a method of treating a subject having a dystrophinopathy disorder requiring a dystrophin gene therapy or a dystrophin protein replacement therapy.

Abstract: Disclosed herein are nucleic acid compositions and methods of use. The nucleic acid compositions may have a therapeutic nucleic acid sequence operably linked to a nuclear targeting sequence that increases expression of the therapeutic nucleic acid in a cell by at least 1.25 fold; at least 80% sequence identity to SEQ ID NO: 6; or a first regulatory element comprising a promoter sequence operably linked to a hemoglobin subunit gamma intron (hBGi) sequence, and a second regulatory element comprising a woodchuck hepatitis posttranscriptional regulatory element (WPRE) sequence. The nucleic acid compositions with these features may enhance therapeutic nucleic acid transfection and expression. Also disclosed herein are transgenes optimized for gene therapy applications, including novel FVIII transgene sequences, in which the B domain may be non-naturally occurring the A1 and/or A3 domain may include at least one amino acid substitution.

Abstract: Provided are systems and methods of delivering an exogenous payload to a cell in a tissue of a subject, including: administering an exogenous payload to the subject; administering a sonoactive agent to the subject, and applying an acoustic radiation force (ARF) to the subject, thereby generating shear waves in the tissue of the subject, wherein the acoustic radiation force enhances delivery of the exogenous payload to the cell in the tissue of an organ of the subject.

Abstract: Provided are methods, compositions, and kits for improving a delivery of a nucleic acid to a cell in an organ of a subject. Such methods, compositions, and kits may include a sonoactive agent and a nucleic acid comprising a cargo polynucleotide and a nuclear localization element and/or an innate immune response avoidance moiety, which may be an aptamer. Delivery may involve sonoporation.

Abstract: A shower curtain having one or more pockets formed near the lower edge and on the side facing the shower nozzle is disclosed. The pockets are designed to collect water while the shower is in use. The pockets have drain holes that allow the water to slowly drain, such that while the shower is in use, the pockets will fill, but when the shower is shut off, the pockets will slowly drain until they are empty. The pockets are in fluid communication with one another by way of openings extending between the pockets. The bottom edge of the pocket is sloped with the highest point of the bottom wall at or near the center of the shower curtain encouraging water to flow toward the edges. Below the pockets, the shower curtain is formed of an air permeable material such as nylon mesh.

Abstract: An ultrasonic imaging agent is formed by subjecting an aqueous protein solution to high frequency sonication while heating the solution sufficiently to denature portions of the protein, thereby forming a dispersion of microbubbles stabilized by denatured protein. In a preferred embodiment, the protein solution is formed from human serum albumin, and the formed microbubbles have a relatively uniform size in the 2 to 5 micron range, suitable for imaging of pulmonary or small organ vasculature.

Abstract: A method of ultrasonic imaging for use in medical procedures is disclosed. The method comprises providing specifically defined microbubbles formed by sonicating a bicompatible liquid comprising a sonicated aqueous protein solution, preferably a 5% solution of human serum albumin, and denaturing the protein therein by heat or chemical methods; injecting the microbubbles into an animal or human to thereby alter the acoustic properties of an area to be imaged; and then ultrasonically scanning the area so as to obtain an ultrasound scanning image.

Abstract: A method of ultrasonic imaging for use in medical procedures is disclosed. The method comprises injecting specifically defined microparticles or sonicated microbubbles into an animal or human to thereby alter the acoustic properties of an area to be imaged, and then ultrasonically scanning the area so as to obtain an image.