Abstract: The present disclosure is in the field of modulation of genes involved in rare diseases including for diagnostics and therapeutics for rare diseases such as Angelman's Syndrome, Facioscapulohumeral Muscular Dystrophy (FHMD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal dementia (FTD) and Spinal Muscular Atrophy (SMA).

Abstract: A pump guard with air deflector, fire protection, to provide an inner housing or guard configured to cover the mechanical seal assembly, the motor coupling, mechanical seals, and part of bearing housing between a drive motor and a fluid pump, where the inner housing or guard includes an extended guards to extend coverage of the inner housing or guard around the fluid pump side of the coupling, the addition of directional deflector configured to focus an air stream directly on the bearing/seal assembly itself, and an outer “Air scoop” housing or guard configured to direct motor induced airflow over the mechanical seal assembly and into directional deflector.

Abstract: Disclosed herein, are compositions and methods useful in expressing a functional NGLY1 protein in a subject by administration of an rAAV containing a transgene encoding NGLY1. Also disclosed herein are methods for treating an NGLY1 gene deficiency in a subject in need thereof.

Abstract: Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic IDUA or IDS protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of MPS I or MPS II disease.

Abstract: Disclosed herein are methods and compositions for targeted, nuclease-mediated insertion of transgene sequences into the genome of a cell.

Abstract: Disclosed herein are methods and compositions for targeted, nuclease-mediated insertion of transgene sequences into the genome of a cell.

Abstract: A pump combination guard and methods of use thereof, having an inner housing or guard configured to cover mechanical seal assembly and the motor coupling housed between a drive motor and a fluid pump to enable foam spray nozzles to be directly inserted into the coupling guard housings for fire suppression and an outer “Air scoop” housing or guard configured to direct motor induced airflow over the mechanical seal assembly thereby enabling fire protection and cooling to extend seal life and to protect mechanical seals from pre-mature failure.

Abstract: Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease.

Abstract: Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease.

Abstract: Disclosed herein are methods and compositions for enhancing insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease or a hemophilia by administering one or more topoisomerases inhibitors, one or more stabilizers of R loop formation or inhibitors of R-loop repair and/or one or more up-regulators of the TC-NER pathway to the target cell.

Abstract: A pump combination guard and methods of use thereof, having an inner housing or guard configured to cover mechanical seal assembly and the motor coupling housed between a drive motor and a fluid pump to enable foam spray nozzles to be directly inserted into the coupling guard housings for fire suppression and an outer “Air scoop” housing or guard configured to direct motor induced airflow over the mechanical seal assembly thereby enabling fire protection and cooling to extend seal life and to protect mechanical seals from pre-mature failure.

Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a cell.

Abstract: Disclosed herein are methods and compositions for targeted, nuclease-mediated insertion of transgene sequences into the genome of a cell.

Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a cell.

Abstract: Disclosed herein are methods and compositions for targeted, nuclease-mediated insertion of transgene sequences into the genome of a cell.

Abstract: The present disclosure is in the field of modulation of genes involved in rare diseases including for diagnostics and therapeutics for rare diseases such as Angelman's Syndrome, Facioscapulohumeral Muscular Dystrophy (FHMD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal dementia (FTD) and Spinal Muscular Atrophy (SMA).

Abstract: Methods and compositions for increasing nuclease-mediated genomic modification using DNA repair inhibitors are provided.

Abstract: Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease.

Abstract: Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease.

Abstract: Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic IDUA or IDS protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of MPS I or MPS II disease.