This invention relates to polynucleotides comprising optimized GALC open reading frame (ORF) sequences, vectors comprising the same, and methods of using the same for delivery of the ORF to a cell or a subject and to treat disorders associated with aberrant expression of a GALC gene or aberrant activity of a GALC gene product in the subject, such as Krabbe disease (i.e., globoid cell leukodystrophy (GLD)).

The present invention provides a delivery technique for delivering a gene modification tool capable of providing a high gene modification efficiency in cells. The composition according to the present invention is a composition for inducing gene modification at a target gene locus in a cell, the composition containing 1) a compound represented by formula (I) or a salt thereof; 2) a structural lipid; and 3) a guide RNA or a DNA including a sequence encoding the guide RNA, and/or an RNA-guided nuclease or a nucleic acid including a sequence encoding the RNA-guided nuclease. In formula (I), n represents an integer of 2 to 5, R represents a linear C.sub.1-5 alkyl group, a linear C.sub.7-11 alkenyl group, or a linear C.sub.11 alkadienyl group, and wavy lines each independently represent a cis-type bond or a trans-type bond.

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The present invention relates to a hemophilia B rat and a method of producing the hemophilia B rat. More particularly, the present invention relates to a hemophilia B rat having F9 factor knocked-down or knocked-out and a method of producing the hemophilia B rat.

The present disclosure provides short enhanced human opsin promoters for rod-specific expression that satisfy a need in the art for promoters that are able to control transgene expression both efficiently and selectively in rod cells, with little to no off-target expression in other photoreceptor cells. The disclosure provides vector constructs comprising transgenes operably controlled by this enhanced human opsin promoter that may be delivered and targeted with high specificity to rod cells. The disclosure also provides compositions comprising these vector constructs and methods for administration of these compositions to subjects in need thereof.

An ergonomic training harness for use with an animal, the harness having a back panel with an outward-facing surface and an inward-facing surface and a chest panel with an outward-facing surface and an inward-facing surface. The harness also includes a chest strap having a first end and a second end, wherein each of the first end and the second end are coupled to the back panel. Additionally, the harness includes an underbelly strap coupled to the back panel, and a longitudinal strap coupled to the chest panel, the longitudinal strap having a first loop portion and a second loop portion. The chest strap is configured to pass through the first loop portion of the longitudinal strap and the underbelly strap is configured to pass through the second loop portion of the longitudinal strap.

The present invention is a system and method for using DNA markers and advances in genetic phenotyping information to obtain canine offspring having a desired phenotype, wherein the genetic information of a canine sire or dam, the known parent, is used in combination with the desired phenotype of the offspring to thereby determine the necessary DNA markers of the unknown canine parent, generating a visual illustration of the unknown canine parent, and then searching through a database of potential sires and dams to identify a dam or sire that most closely resembles the predicted unknown canine parent.

Provided herein are multivalent particles and compositions of multivalent particles for blocking viral infection.

The disclosure describes methods of treating humans with Duchenne muscular dystrophy by providing doses of an AAV9 vector that expresses a mini-dystrophin protein in transduced muscle cells.

Disclosed is CD31.sup.shed for use as a molecular imaging target in the molecular imaging of an inflammatory condition. Administering the radiolabeled peptide P8RI as CD31.sup.shed ligand in different rat models of inflammation indeed showed that CD31.sup.shed is present on activated cells in a quantity allowing a detectable signal, whereas the noise signal corresponding to CD31.sup.shed present on activated circulating cells and on other organs or cells not involved in inflammation was little. Also disclosed is a labeled CD31.sup.shed ligand and the use thereof as a molecular imaging agent in the molecular imaging of an inflammatory condition. The molecular imaging of inflammatory sites particularly allows determining whether a subject suffers from or is at risk of having an inflammatory condition or is at risk of recurrence of an inflammatory condition after an anti-inflammatory treatment.

Compositions and methods for insulin sensitization are provided.