Provided herein are engineered nucleic acids (e.g., expression vectors, including viral vectors, such as lentiviral vectors, adenoviral vectors, AAV vectors, herpes viral vectors, and retroviral vectors) that encode OCT4; KLF4; SOX2; or any combination thereof that are useful, for example, in inducing cellular reprogramming, tissue repair, tissue regeneration, organ regeneration, reversing aging, or any combination thereof. Also provided herein are recombinant viruses (e.g., lentiviruses, alphaviruses, vaccinia viruses, adenoviruses, herpes viruses, retroviruses, or AAVs) comprising the engineered nucleic acids (e.g., engineered nucleic acids), engineered cells, compositions comprising the engineered nucleic acids, the recombinant viruses, engineered cells, engineered proteins, chemical agents that are capable of activating expression of OCT4; KLF4; SOX2; or any combination thereof, an engineered protein selected from the group consisting of OCT4; KLF4; SOX2; or any combination thereof, an antibody capable of activating expression of OCT4; KLF4; SOX2; or any combination thereof, and methods of treating a (e.g., ocular disease), preventing a disease (e.g., ocular disease), regulating (e.g., inducing or inducing and then stopping) cellular reprogramming, regulating tissue repair, regulating tissue regeneration, or any combination thereof).

The present invention provides a composition comprising fetal cartilage tissue-derived cells and a fetal cartilage tissue-derived extracellular matrix as active ingredients for regenerating a growth plate. The composition for regenerating a growth plate can inhibit bone bridge formation in a growth plate injury region without a scaffold and differentiate to a growth plate cartilage tissue to effectively fill and regenerate the injured region therewith, whereby the regenerated growth plate tissue can recover growth ability. In addition, the composition is compatible with and safe to biological tissues and is characterized by high reproducibility and homogeneity.

The present invention provides a composition comprising fetal cartilage tissue-derived cells and a fetal cartilage tissue-derived extracellular matrix as active ingredients for regenerating a growth plate. The composition for regenerating a growth plate can inhibit bone bridge formation in a growth plate injury region without a scaffold and differentiate to a growth plate cartilage tissue to effectively fill and regenerate the injured region therewith, whereby the regenerated growth plate tissue can recover growth ability. In addition, the composition is compatible with and safe to biological tissues and is characterized by high reproducibility and homogeneity.

Provided is an immunostimulatory composition, comprising a saponin and a CpG oligodeoxynucleotide, or consisting of an adjuvant comprising a saponin and a CpG oligodeoxynucleotide, wherein the sequence of the CpG oligodeoxynucleotide has two or more copies of 5′-TTCGTT-3′ motif or 5′-TCGTCGTCG-3′ motif. Also provided is use of the immunostimulatory composition in the preparation of a medication for treating diseases.

The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A.

The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A.

The use of markers that participate in inflammatory processes and are associated cytokines in the diagnosis, treatment or prophylaxis of diseases is disclosed. Specifically, cytokines are used to diagnose or treat non-neoplastic or non-leukaemic diseases such as autoimmune diseases or neurodegenerative disorders by the process of taking a DNA bearing sample from a subject animal and analysing the sample to determine the allelic variants present at one or more of the SNP loci at positions −1082, −819 and −592 of the gene encoding IL-10. A method of treating Alzheimer's disease, autoimmune diseases or other neurodegenerative disorders is disclosed by modulating, that is augmenting or decreasing, the function of a gene having one of the allelic polymorphisms of IL-10. IL-6 inhibitors and IL-10 promoters can be used in the manufacture of a medicament for the treatment of prophylaxis of Alzheimer's disease.

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of signal-sensor polynucleotides, primary transcripts and mmRNA molecules.

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of signal-sensor polynucleotides, primary transcripts and mmRNA molecules.

The invention provides methods of treating an allergic inflammatory condition characterized by inflammation of the squamous epithelium of a target tissue by administering to the subject a pharmaceutical composition comprising a kallikrein 5 (KLK5) inhibitor, and a pharmaceutically acceptable carrier.