An isolated nucleic acid encoding a monovalent antibody fragment comprising a first polypeptide comprising a light chain variable domain and two constant domains and a second polypeptide comprising a heavy chain variable domain and two constant domains, wherein two chain constant domains are light chain constant domains and two constant domains are CH1 heavy chain constant domains.

A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).

The present disclosure provides codon optimized Factor VIII sequences, vectors, and host cells comprising codon optimized Factor VIII sequences, polypeptides encoded by codon optimized Factor VIII sequences, and methods of producing such polypeptides. The present disclosure also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject a codon optimized Factor VIII nucleic acid sequence or the polypeptide encoded thereby.

Embodiments of the disclosure concern methods and compositions for treatment of a cardiac-related medical condition. In particular embodiments, ANGPTL8 is provided to an individual in need thereof for the treatment or prevention of a cardiac-related medical condition. In certain cases, ANGPTL8 is provided to an individual with cardiomyopathy, such as Adriamycin-induced cardiomyopathy, using ultrasound-targeted microbubble destruction.

Provided herein are methods, systems, and related vectors and compositions allowing for noninvasive control of neural circuits. In particular, the methods and systems herein described utilize acoustically targeted chemogenetics to achieve a noninvasive neuromodulation in specifically selected cell-types among spatially selected brain regions.

The present invention relates to methods and compositions for inhibiting metastatic spread of cancer and/or inhibiting progression of pre-existing metastatic disease in a subject using L1CAM inhibition.

The present invention provides a method for assessing the risk of cancer in a subject by detecting elevated methylation level in the genomic sequence of the TET1 gene, which leads to suppressed expression of this gene. A kit and device useful for practicing such a method are also provided.

A method of treating hyperglycemia, diabetes, metabolic syndrome, insulin resistance (insulin insensitivity), impaired glucose tolerance, high glucose levels, pulmonary hypertension, and/or a condition arising from any of the foregoing in a patient is provided. The method comprises knocking down mARC2 or mARC1 expression in the patient, or otherwise decreasing mARC2 and mARC1 activity in the patient.

Pharmaceutical compositions and methods of making the composition are disclosed, wherein nucleic acid-based drugs can be introduced into cells by using cell-derived natural or artificial nanovesicles loaded with nucleic acid-based drugs. In the compositions, natural or artificial nanovesicles loaded with antisense oligonucleotides exhibit properties that inhibit proliferation and migration of cancer cells and increase drug sensitivity when co-administered with conventional chemotherapeutic agents.

Provided are methods and compositions for treating cancer in a subject in need thereof. One of the top gene products in glioblastoma multiforme (GBM) is KLRB1 (also known as CD161), a C-type lectin protein that binds to CLEC2D. Binding of CLEC2D to the KLRB1 receptor inhibits the cytotoxic function of NK cells as well as cytokine secretion. KLRB1 is only expressed by small subpopulations of human blood T cells, and consequently little is known about the function of this receptor in T cells. However, preliminary data demonstrate that KLRB1 expression is induced in T cells within the GBM microenvironment. In an exemplary embodiment, a method is provided comprising administering an agent capable of blocking the interaction of KLRB1 with its ligand. The agent may comprise an antibody or fragment thereof, which may bind KLRB1 or CLEC2D.