Abstract: In certain aspects, the present disclosure provides dosing regimens, dosing forms, and formulations comprising a recombinant fusion protein comprising human activin receptor type-II (ActRII) polypeptides or derivatives thereof linked to a constant domain of an immunoglobulin, such as human IgG1 Fc domain. The disclosure also provides kits and methods for using such formulations to treat a human subject with a thalassemia or myelodysplastic syndromes (MDS).

Abstract: The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, comprising recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Sf9 insect cells as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and Baculoviral Infected Insect Cells (BIICs) in the production of AAV particles.

Abstract: The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles and AAV formulations, including recombinant adeno-associated virus (rAAV) particles and formulations. In certain embodiments, the present disclosure presents methods and systems for clarifying, purifying, formulating, filtering and processing AAV particles and AAV formulations. The present disclosure also describes compositions, methods and processes for the design, preparation, manufacture, use and/or formulation of AAV particles comprising modulatory polynucleotides, e.g., polynucleotides encoding small interfering RNA (siRNA) molecules which target the Huntingtin (HTT) gene (e.g., the wild-type or the mutated CAG-expanded HTT gene). Methods for using formulated AAV particles comprising modulatory polynucleotides to inhibit the HTT gene expression in a subject with a neurodegenerative disease (e.g., Huntington's Disease (HD)) are also disclosed.

Abstract: The present disclosure is directed to parvovirus genomes; plasmid vectors encoding parvovirus genomes, and particles and populations thereof; as well as methods of their production and use.

Abstract: Novel polynucleotides and the proteins encoded thereby are disclosed.

Abstract: Novel polynucleotides and the proteins encoded thereby are disclosed.

Abstract: Novel polynucleotides and the proteins encoded thereby are disclosed.

Abstract: Novel polynucleotides and the proteins encoded thereby are disclosed.

Abstract: Remote supply circuitry for remotely supplying a subscriber connections in a communications system wherein subscribers can be connected to a central point via subscriber lines. Subscriber supply voltage or power supply is provided for each subscriber via a supply voltage source assigned to the subscriber and at least one supply isolating element wich is connected in series with the subscriber and associated subscriber line. The supply voltage source assigned to the subscriber supplies an output voltage which is controllable as a function of a measurable variable.