A fusion protein according to an embodiment of the present disclosure includes a growth differentiation factor 11 and an epidermal growth factor with an enhanced anti-oxidation activity and an enhanced skin cell proliferation effect, A cosmetic composition according to an embodiment of the present disclosure includes the fusion protein as an effective component. The cosmetic composition can be advantageously used in future as a material of a functional cosmetic product.

Disclosed herein are nucleic acid-guided nucleases, guide nucleic acids, and targetable nuclease systems, and methods of use. Disclosed herein are engineered non-naturally occurring nucleic acid-guided nucleases, guide nucleic acids, and targetable nuclease systems, and methods of use. Targetable nuclease systems can be used to edit genetic targets, including recursive genetic engineering and trackable genetic engineering methods.

The present invention provides novel expression cassettes, retroviral plasmids, vectors, virions, compositions and recombinant cells comprising a promoter operably linked to a codon optimised recombination activating (RAG1) transgene. These novel expression cassettes, retroviral plasmids, vectors, virions, compositions and recombinant cells are useful in the treatment of diseases caused by complete or partial loss-of-function of the protein encoded by the rag-1 gene, such as RAG-deficient severe combined immunodeficiency (RAG1-SCID), Omenn Syndrome (OS), atypical-SCID or combined immunodeficiency (CID). Corresponding methods of treatment are also provided.

Aspects of the disclosure relate to compositions and methods for expressing one or more Ganglioside GM3 synthase (GM3S) isoforms in a cell or subject. In some aspects, the disclosure relates to methods for treating GM3 synthase deficiency in a subject in need thereof.

In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA protein, which is the E1-alpha subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, in a subject. In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHB protein, which is the E1-beta subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, in a subject. In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA and BCKDHB proteins, in a subject. In some embodiments, the disclosure provides methods of treating a subject having Maple Syrup Urine Disease (MSUD).

Carbamoyl phosphate synthetase 1 (CPS1) deficiency is a metabolic disorder of the liver that results m abnormal nitrogen metabolism. To illustrate the ability of gene therapy to treat CPS1 deficiency, two adeno-associated viruses encoding portions of a codon optimized CPS1 were generated and tested in a conditional CPS1 knock out mouse model. When administered to mice having knocked out endogenous CPS1 expression, mice from this model demonstrate homologous recombination and reconstitution of the codon optimized CPS1 gene, expression of the CPS1 protein and the associated control of plasma ammonia following the administered AAVs comprising the CPS1 gene sequences. While all control mice perish, the mice in this model live and have normal behavior. As there is no effective therapy for human patients with the CPS1 disorder, this invention can address this unmet need for these patients.

Provided is an invention based, in part, on novel gene constructs that encode a microdystrophin protein for use in gene therapy. The microdystrophin gene constructs and expression cassettes were engineered for improved therapy with respect to efficacy, potency and safety to the subject when expressed by a viral vector in muscle cells and/or CNS cells.

The present invention provides, among other things, improved pharmaceutical compositions comprising codon-optimized mRNA encoding a peptide or polypeptide encapsulated in a lipid nanoparticle comprising one or more of the cationic lipids that are particularly effective for pulmonary delivery.

The present invention relates to a vaccine composition for preventing severe acute respiratory syndrome coronavirus 2 infection. The vaccine composition for preventing severe acute respiratory syndrome coronavirus 2 infection according to the present invention comprises spike and ORF3a or nucleocapsid as an antigen against SARS-CoV-2. The vaccine composition has a significant effect of inducing antibody immune responses and T-cell immune responses by a plurality of co-expressed antigens compared to those comprising one antigen, and thus can be variously used in the field of prevention of severe acute respiratory syndrome coronavirus 2 infection.

Disclosed herein is a method for expressing phytase in a filamentous fungus by using an optimized Escherichia coli phytase gene having a nucleotide sequence as shown in SEQ ID NO. 7 and a signal peptide having a nucleotide sequence as shown in SEQ ID NO. 12.