Abstract: Aspects of the application provide anti-hemojuvelin antibodies and methods of using the same in treating myelofibrosis and/or conditions associated with myelofibrosis.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

Abstract: Aspects of the application provide anti-hemojuvelin antibodies and methods of using the same in treating anemias of chronic disease, iron-restricted anemias, and/or conditions associated with anemia.

Abstract: Aspects of the application provide anti-hemojuvelin antibodies and methods of using the same in treating myelofibrosis and/or conditions associated with myelofibrosis.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result,

Abstract: Aspects of the application provide hepcidin antagonists and methods of using the same in treating myelofibrosis and/or conditions associated with myelofibrosis. In certain embodiments, methods are provided for treating myelofibrosis, which is generally characterized as a myeloproliferative disease associated with chronic inflammation and progressive marrow fibrosis. Anemia is a major clinical problem in myelofibrosis and is associated with negative outcomes. Such anemia is generally caused by, or associated with, bone marrow failure, splenomegaly and/or functional iron deficiency, which may contribute to inflammation.

Abstract: Aspects of the application provide hepcidin antagonists and methods of using the same in treating anemias of chronic disease, iron-restricted anemias, and/or conditions associated with anemia. Accordingly, aspects of the present disclosure relate to treating a subject with high hepcidin levels (e.g., ACD) by inhibiting the BMP signaling pathway (e.g., hemojuvelin-induced BMP signaling pathway) and/or the inflammatory response (e.g., IL-6 mediated inflammatory pathway).

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

Abstract: The present invention provides a method of treating a disease or disorder caused by and/or associated with CSF1R dysfunction in a human patient, the method comprising administering to the patient in need thereof an effective amount of a compound that increases the activity of triggering receptor expressed on myeloid cells 2 (TREM2). In some embodiments, compound that increases the activity of TREM2 is an agonist of TREM2. In some embodiments, the agonist of TREM2 is a small molecule agonist of TREM2 or an antibody agonist of TREM2.

Abstract: The present invention relates to methods and compositions for editing a SERPINA1 polynucleotide, e.g., a SERPINA1 polynucleotide comprising a SNP associated with alpha 1 antitrypsin deficiency. The invention also relates to methods and compositions for treating or preventing alpha 1 antitrypsin deficiency in a subject.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

Abstract: The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.