In certain embodiments, the disclosure relates to the polynucleotide sequences of respiratory syncytial virus (RSV). In certain embodiments, the disclosure relates to isolated or recombinant nucleic acids and polypeptides comprising desirable nucleic acid sequences and mutations disclosed herein. In certain embodiments, isolated or recombinant RSV comprising the nucleic acids and polypeptides disclosed herein (e.g., attenuated recombinant RSV) are also provided, as are immunogenic compositions including such nucleic acids, polypeptides, and RSV genomes that are suitable for use as vaccines. Attenuated or killed RSV containing these nucleic acids and mutation in the form of copied nucleic acids (e.g., cDNAs) are also contemplated.

The present invention relates to a nucleic acid expression cassette, in particular for the expression of a human liver-specific and/or liver-expressed protein and/or preferably physiologically active domains and/or fragments thereof in a patient suffering from a monogenetic disorder caused by a mutation in the gene coding for the liver-specific and/or liver-expressed protein.

Described are recombinant nucleic acid molecules for increased expression of Cas9 in human liver. In some embodiments, the recombinant nucleic acid molecules are provided in compositions and methods for gene editing, specifically using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR).

Provided herein are compositions and methods related to a multivirus-specific T cell immunotherapy.

Provided herein are gene therapy methods for the treatment of mucopolysaccharidosis type IV A (MPS IV A) involving the use of recombinant adeno-associated viruses (rAAVs) to deliver human N-acetylgalactosamine-6-sulfate sulfatase (hGALNS) to the bone of a human subject diagnosed with MPS IVA. Also provided herein are rAAVs that can be used in the gene therapy methods and methods of making such rAAVs.

The disclosure relates to nucleic acid expression cassettes for the treatment of neurodegenerative disorders. Methods of treating neurodegenerative disorders such as Alzheimer's disease, frontotemporal dementia, frontotemporal lobar degeneration, Pick's disease, Lewy body dementia, memory loss, cognitive impairment, and mild cognitive impairment are also provided.

Synthetic polynucleotides encoding human propionyl-CoA carboxylase beta (synPCCB) and exhibiting augmented expression in cell culture and/or in a subject are described herein. An adeno-associated viral (AAV) gene therapy vector encoding synPCCB successfully rescued the neonatal lethal phenotype displayed by propionyl-CoA carboxylase beta (Pccb.sup.−/−) deficient mice, lowered circulating methylcitrate levels in the treated animals, and resulted in prolonged hepatic expression of the product of the synPCCB transgene in vivo.

Severe Acute Respiratory Syndrome Coronavirus 2 antigen virus-like particles (VLPs) and recombinant immune complexes (RICs) are described, along with methods of making said VLPs and RICs in plants and using said VLPs and RICs to induce an immune response in a subject.

The present disclosure provides codon optimized nucleotide sequences encoding human REP1, vectors, and host cells comprising codon optimized REP1 sequences, and methods of treating retinal disorders such as choroideremia comprising administering to the subject a codon optimized sequence encoding human REP1.

Provided is an antigen including a recombinant respiratory syncytial virus (RSV) F protein, wherein the recombinant RSV F protein includes an antigenic region flanked with an HRN region and an HRC region, and the antigenic region includes one or more antigenic sites selected from the group consisting of site Ø, site II, and site IV. The present disclosure also provides a nucleic acid molecule encoding the antigen and a vaccine composition including the antigen for eliciting an immune response against RSV.