Provided is a recombinant adeno-associated virus (rAAV) having an AAV capsid and a vector genome which comprises a nucleic acid sequence encoding a functional CDKL5 (hCDKLK5). Also provided are a production system useful for producing the rAAV, a pharmaceutical composition comprising the rAAV, and a method of treating a subject having CDD, or ameliorating symptoms of CDD, or delaying progression of CDD via administrating an effective amount of the rAAV to a subject in need thereof.

The present disclosure generally relates to polynucleotides and AAV vectors that provide for the expression of ALD protein in target (e.g., neurons or glial) cells when administered to subjects in need thereof. The present disclosure further relates to compositions comprising such a polynucleotide or vector. These polynucleotides, vectors, and compositions may be used for the treatment and prevention of ALD or AMN in subjects in need thereof.

Disclosed are invariant natural killer T (iNKT) cells engineered using hematopoietic stem and progenitor cells (HSPCs) and methods of making and using thereof. Specifically, the engineered cells iNKT are genetically modified to contain at least one exogenous invariant natural killer T cell receptor (iNKT TCR) nucleic acid molecule. Further disclosed are iNKT TCR nucleotide sequences and codon optimized sequences for expression.

Provided herein are recombinant nucleic acids and viral vectors thereof encoding a SARS-CoV-2 spike protein that have been optimized for expression in mammalian cells.

A composition for treating cancer is disclosed. The composition includes a lentiviral particle and an aminobisphosphonate drug. The lentiviral particle is capable of infecting a target cell, such as a cancer cell, and includes an envelope protein optimized for targeting such target cell and a viral vector. The viral vector includes a small RNA optimized to target an FDPS mRNA sequence. The aminobisphosphonate drug includes zoledronic acid.

Adeno-associated virus rh.20 sequences, vectors containing same, and methods of use are provided.

The present invention relates to a porcine alpha-S1-casein gene, a porcine alpha-S1-casein gene promoter, an expression comprising the same promoter, and a method for the production of a target protein using the same expression vector. The promoter of the present invention facilitates the mammary gland-specific expression of the target protein. Accordingly, an animal transformed with the promoter secretes the target protein in milk at high concentration, and thus can be advantageously used for the production of useful proteins.

The invention provides improved compositions for adoptive T cell therapies for B cell related conditions.

Described herein are methods and compositions related to GATA-1 gene therapy for the treatment of Diamond-Blackfan anemia.

The present invention relates to a Crumbs homologue (CRB) therapeutic for use as a medicament or in a method of treatment or prophylaxis, for example in the treatment or prophylaxis of a retinal disorder due to mutations in the Crumbs homologue-1 (CRB1) gene, such as Leber's congenital amaurosis 8 (LCA8) or retinitis pigmentosa 12 (RP12). In particular, the present invention relates to a recombinant viral vector comprising CRB2 or modified non-toxic forms of either CRB1 or CRB3 that resemble CRB2.