The invention solves the problem of providing new therapies that are effective in the treatment of muscular dystrophies through the use of compositions comprising a compound capable of reducing the expression of miRNA-106b in muscle satellite stem cells of a human or animal subject with respect to the expression observed in the absence of the compound in said cells.

A retroviral construct harboring nucleic acids encoding a high mobility group AT-hook 2 (HMGA2) protein and lacking let-7 binding sites is described as are methods of using the retroviral vector to increase the efficacy and in vivo expansion of transduced cells in gene therapy applications.

Provided herein are methods and related compositions for administering viral transfer vectors and antigen-presenting cell targeted immunosuppressants.

Provided herein are IL-21 muteins and fusion proteins comprising the same for use in methods of treating a disease. Related conjugates, nucleic acids, vectors, host cells, pharmaceutical compositions and kits are also provided herein. Methods of making the IL-21 muteins and fusion proteins comprising the same, as well as methods of treating a subject in need thereof, are provided by the present disclosure. Further provided are PD-1 antigen-binding proteins.

Provided herein are methods and related compositions for administering viral transfer vectors and antigen-presenting cell targeted immunosuppressants.

Methods and compositions are provided for producing synthetically-derived keratin compositions and keratin products.

Provided herein are methods and related compositions for administering viral transfer vectors and antigen-presenting cell targeted immunosuppressants.

Disclosed herein are viral vectors for use in recombinant molecular biology techniques. In particular, the present disclosure relates to self-limiting viral vectors comprising genes encoding site-specific endonucleases as well as recognition sequences for site-specific endonucleases such that expression of the endonuclease in a cell cleaves the viral vector and limits its persistence time. In some embodiments, the viral vectors disclosed herein also carry directives to delete, insert, or change a target sequence.

The present invention relates to a new binder molecule useful e.g. as an immune suppressor. In particular, it has been recognized that the UL11 protein of human the cytomegalovirus, binds to the CD45 molecule, potentially altering the immune system of an individual. In addition, the present invention relates to binder molecules, in particular, fusion molecules containing the CD45 binding domain of the UL11 protein in combination with a second molecule of interest for delivery of said molecule to cells expressing the CD45 molecule. Moreover, the present invention relates to pharmaceutical compositions comprising the UL11 protein of human cytomegalovirus, or derivatives or homologs thereof, or a nucleic acid sequence encoding the same.

Methods are provided for the prognosis, diagnosis and treatment of various pathological states, including cancer, chemotherapy resistance and dementia associated with Alzheimer's disease. The methods provided herein are based on the discovery that various proteins with a high level of sialylation are shown herein to be associated with disease states, such as, cancer, chemotherapy resistance and dementia associated with Alzheimer's disease. Such methods provide a lysosomal exocytosis activity profile comprising one or more values representing lysosomal exocytosis activity. Also provided herein, is the discovery that low lysosomal sialidase activity is associated with various pathological states. Thus, the methods also provide a lysosomal sialidase activity profile, comprising one or more values representing lysosomal sialidase activity. A lysosomal sialidase activity profile is one example of a lysosomal exocytosis activity profile.