Provided herein are methods, compounds, and compositions for reducing expression of sodium voltage-gated channel alpha subunit 2 (SCN2A) in a subject. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a sodium voltage gated channel alpha subunit 1 (SCN1A) related disease or disorder (e.g., Dravet syndrome) in a subject in need.

The present disclosure relates to genetically modified non-human animals that express a human or chimeric (e.g., humanized) CD3e (T-cell surface glycoprotein CD3 epsilon chain), and methods of use thereof.

The present invention provides methods, agents, compounds, and compositions useful for administering to subjects having or at risk of having anemias. In certain embodiments, methods herein comprise administering two agents to a subject, which are useful for the treatment of an anemia.

The present invention relates to a medical use of Cellular Repressor of E1A-stimulated Genes (CREG), in particular to a use of CREG protein or active fragment thereof in manufacture of a medicament for preventing and/or treating myocardial infarction, a use in manufacture of a medicament for preventing and/or treating ventricular remodeling after myocardial infarction and heart failure after myocardial infarction, a use in manufacture of a medicament for prevention and/or treatment of myocardial ischemia-reperfusion injury, a use in manufacture of a medicament for prevention and/or treatment of salt-sensitive hypertensive myocardial fibrosis.

The present disclosure relates to genetically modified non-human animals that express a human or chimeric (e.g., humanized) LAG3, and methods of use thereof.

The present disclosure relates to genetically modified non-human animals that express a human or chimeric (e.g., humanized) CD3e (T-cell surface glycoprotein CD3 epsilon chain), and methods of use thereof.

The present invention relates to recombinant laminin adeno-associated viral vector (AAV) constructs and related methods for restoring laminin expression in deficient mammals, or in mammals with basement membrane instability.

Selectively providing voltage-gated sodium channel function sufficient to rescue impaired Nav1.1 function to inhibitory neurons is described. Provided voltage-gated sodium channel function sufficient to rescue impaired Nav1.1 function in inhibitory neurons can be used to treat disorders such as epilepsy, and more particularly, Dravet Syndrome.

Described herein is the discovery that neither the nuclear localization signal (NLS) nor the prion-like domain (PLD) of TDP-43 is necessary for embryonic stem cell culture and differentiation into motor neurons in vitro. The ability of ES cells to express these TDP-43 mutants and differentiate into motor neurons that exhibit an ALS-like phenotype whereby the TDP-43 mutants redistribute to and aggregate in the cytoplasm and fail to regulate cryptic exon splicing allows these cells to act as a model of TDP-43 proteinopathy for the testing of candidate therapeutic agents that may resolve such proteinopathy. Additionally, these ES cells may be used to successfully generate non-human animals, e.g., mice, that also exhibit hallmark symptoms of ALS and that may be used in testing candidate agents useful in treating TDP-43 proteinopathies.

This disclosure relates to a rodent model. More specifically, this disclosure relates to a loss of function of solute carrier 39 member 5 (SLC39A5) rodent model. In particular, disclosed herein are genetically modified rodent animals that carry a loss of function mutation in an endogenous Slc39a5 gene and use of such rodent animals in elucidating the role of SLC39A5 in zinc homeostasis, glycemic regulation and lipid metabolism.