Abstract: The present disclosure provides methods of treating subjects having an immune disorder by administering a therapeutically effective amount of an Endoplasmic Reticulum Aminopeptidase 2 (ERAP2) inhibitor to the subject, and optionally an Endoplasmic Reticulum Aminopeptidase 1 (ERAP1) agonist or inhibitor and/or an HLA-Aw19 inhibitor, and also provides methods of identifying subjects having an increased risk for developing an MHC-I-opathy.

Abstract: Nucleic acid constructs and compositions that allow insertion and/or expression of a retinoschisin coding sequence are provided. Nuclease agents targeting RSI loci are provided. Compositions and methods of using such constructs for integration into a target genomic locus and/or expression in a cell are also provided. Methods of treating X-linked juvenile retinoschisis using the nucleic acid constructs and compositions are also provided.

Abstract: Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized MYOC locus and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized MYOC locus express a human myocilin protein or a chimeric myocilin protein, fragments of which are from human myocilin. Methods are provided for using such non-human animals comprising a humanized MYOC locus to assess in vivo efficacy of human-myocilin-targeting reagents and reagents for treating glaucoma.

Abstract: Nucleic acid constructs and compositions that allow insertion and/or expression of a retinoschisin coding sequence are provided. Nuclease agents targeting RS1 loci are provided. Compositions and methods of using such constructs for integration into a target genomic locus and/or expression in a cell are also provided. Methods of treating X-linked juvenile retinoschisis using the nucleic acid constructs and compositions are also provided.

Abstract: The present disclosure provides methods of treating subjects having an immune disorder by administering to the subject a therapeutically effective amount of an Endoplasmic Reticulum Aminopeptidase 2 (ERAP2) inhibitor in combination with an HLA-A29 or HLA-B27 inhibitory nucleic acid molecule.

Abstract: Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized TRKB locus and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized TRKB locus express a human TRKB protein or a chimeric transthyretin protein, fragments of which are from human TRKB. Methods are provided for using such non-human animals comprising a humanized TRKB locus to assess in vivo efficacy of human-TRKB-targeting reagents such as nuclease agents designed to target human TRKB.

Abstract: The present disclosure provides methods of treating subjects having an immune disorder by administering to the subject a therapeutically effective amount of an Endoplasmic Reticulum Aminopeptidase 2 (ERAP2) inhibitor in combination with an HLA-A29 or HLA-B27 inhibitory nucleic acid molecule.

Abstract: Disclosed are devices and methods for facilitating directed delivery of a medicament into a human organ of a patient. An apparatus to facilitate directed delivery of a medicament into a human organ of a patient may include a container configured to enclose the medicament, a needle having a passage therethrough and a sharp distalmost tip, and a needle guard at least partially surrounding a shaft of the needle. The needle guard may include a distal surface, wherein the sharp distalmost tip of the needle may be configured to extend through an opening in the distal surface. The needle guard may be moveable relative to the needle along an axis of the needle to control a distance from the distal surface to the distalmost tip of the needle.

Abstract: The present disclosure provides methods of treating subjects having uveitis, and methods of identifying subjects having an increased risk of developing uveitis.

Abstract: Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized MYOC locus and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized MYOC locus express a human myocilin protein or a chimeric myocilin protein, fragments of which are from human myocilin. Methods are provided for using such non-human animals comprising a humanized MYOC locus to assess in vivo efficacy of human-myocilin-targeting reagents and reagents for treating glaucoma.

Abstract: The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting ANGPTL7. The invention also relates to methods of using such dsRNA compositions to inhibit expression of ANGPTL7 and to methods of treating ANGPTL7-associated disorders, e.g., glaucoma, using such dsRNA compositions.

Abstract: The present disclosure provides methods of treating subjects having uveitis, and methods of identifying subjects having an increased risk of developing uveitis.

Abstract: Non-human animals suitable for use as animal models for Retinoschisis are provided. In some embodiments, provided non-human animals are characterized by a disruption in a Retinoschisin-1 locus. In some embodiments, provided non-human animals are characterized by a mutant Retinoschisin-1 gene. The non-human animals may be described, in some embodiments, as having a phenotype that includes the development of one or more symptoms or phenotypes associated with Retinoschisis. Methods of identifying therapeutic candidates that may be used to prevent, delay or treat Retinoschisis or eye-related diseases, disorders or conditions are also provided.

Abstract: Methods of treating B27-negative subjects having uveitis with an Inositol Polyphosphate Multikinase (IPMK) therapeutic agent and/or an Indoleamine 2,3-dioxygenase 2 (IDO2) agonist, and methods of identifying subjects having an increased risk of developing uveitis are described herein.

Abstract: The present disclosure provides methods of treating subjects having inflammation with an Angiopoietin-Like 7 (ANGPTL7) inhibitor and a glucocorticoid, methods of decreasing glucocorticoid-induced ophthalmic conditions in subjects, and methods of identifying subjects having an increased risk of developing glucocorticoid-induced ophthalmic conditions.

Abstract: The present disclosure provides methods of treating subjects having inflammation with an Angiopoietin-Like 7 (ANGPTL7) inhibitor and a glucocorticoid, methods of decreasing glucocorticoid-induced ophthalmic conditions in subjects, and methods of identifying subjects having an increased risk of developing glucocorticoid-induced ophthalmic conditions.

Abstract: Disclosed are devices and methods for facilitating directed delivery of a medicament into a human organ of a patient. An apparatus to facilitate directed delivery of a medicament into a human organ of a patient may include: a needle with a sharp distalmost tip; a needle hub connected to a proximal end of the needle; and an adaptor surrounding at least a portion of the needle; wherein the sharp distalmost tip may be configured to move from a retracted position in which the sharp distalmost tip is within the adaptor to a deployed position in which the sharp distalmost tip protrudes from the adaptor.

Abstract: The present disclosure provides methods of treating patients having an ophthalmic condition, methods of identifying subjects having an increased risk of developing an ophthalmic condition, methods of detecting human angiopoietin like 7 (ANGPTL7) variant nucleic acid molecules and variant polypeptides, and ANGPTL7 variant nucleic acid molecules and variant polypeptides.

Abstract: The present disclosure provides methods of treating patients having an ophthalmic condition, methods of identifying subjects having an increased risk of developing an ophthalmic condition, methods of detecting human angiopoietin like 7 (ANGPTL7) variant nucleic acid molecules and variant polypeptides, and ANGPTL7 variant nucleic acid molecules and variant polypeptides.

Abstract: The present disclosure provides methods of treating subjects having uveitis, and methods of identifying subjects having an increased risk of developing uveitis.