The present disclosure provides production systems and host cells to produce collagen 7 compositions comprising recombinant collagen 7 and/or functional variants thereof. The host cells are genetically engineered to stably express rCol7 and functional variants thereof. The collagen 7 composition can be used to restore collagen 7 levels in a subject in need, and for preventing, preventing the progression of, alleviating, and delaying the on-set of a skin condition, e.g., skin wound associated with dystrophic epidermolysis bullosa (DEB).

The present invention relates to, inter alia, an engineered cell (e.g., iPSC, IPS-derived NK, or NK cell) comprising a disrupted B2M gene and an inserted polynucleotide encoding one or more of SERPINB9, a fusion of IL15 and IL15Rα, and/or HLA-E. The engineered cell can further comprise a disrupted CIITA gene and an inserted polynucleotide encoding a CAR, wherein the CAR can be an anti-BCMA CAR or an anti-CD30 CAR. The engineered cell may further comprise a disrupted ADAM17 gene, a disrupted FAS gene, a disrupted CISH gene, and/or a disrupted REGNASE-1 gene. Methods for producing the engineered cells are also provided, and therapeutic uses of the engineered cells are also described. Guide RNA sequences targeting described target sequences are also described.

Provided are compositions and methods for introducing proteins into cells. The compositions and methods relate to introducing a foreign protein as an engineered component of a paramyxovirus virus like particle (VLP). The compositions and methods pertain to modified VLPs that contain a contiguous recombinant polypeptide comprising i) all or a segment of a C-terminal domain of a paramyxovirus nucleocapsid protein and ii) a polypeptide sequence of a distinct protein that is an enzyme such as a recombinase.

Provided are modified virus-like particles (VLPs) of paramyxoviruses, compositions containing them, methods of using the VLPs for delivery of any particular protein of interest to any of a variety of cells, kits that contain expression vectors for making, using and detecting VLPs, and methods for screening for anti-viral compounds using the VLPs. The modified VLPs contain a contiguous recombinant polypeptide that contains i) all or a segment of a C-terminal domain of a paramyxovirus nucleocapsid protein and ii) a polypeptide sequence of a distinct protein. Non-covalent complexes of paramyxovirus M protein and fusion proteins that contain a C-terminal domain of a paramyxovirus nucleocapsid protein and a polypeptide sequence of a distinct protein are provided, as are non-covalent complexes of cells, and cell receptors, with modified VLPs.

Molecular determinants of cancer response to immunotherapy are described, as are systems and tools for identifying and/or characterizing cancers likely to respond to immunotherapy.

The methods and compositions described herein address the need in the art by providing peptides and polypeptides comprising a growth factor binding domain. In some embodiments, the peptides have an amino acid sequence that is at least 80% identical to one of SEQ ID NOS:1-7, 13-15, 49-50, or 66-70, or a fragment thereof; wherein the peptide is less than 300 amino acids in length.

Use of human milk oligosaccharide LNT, for increasing serpin protein production in Bifidobacterium longum subsp. longum.

Disclosed herein are SERPIN peptides, and analogues and derivatives thereof, and uses of the same for treating various conditions associated with LRP1 mediation.

Described herein are compositions and methods for treating a subject having or at risk of developing hereditary angioedema. Using the compositions and methods of the disclosure a patient may be provided one or more agents that elevate the expression and/or activity levels of C1-esterase inhibitor (C1-INH). Exemplary agents that may be used in conjunction with the compositions and methods of the disclosure for this purpose include cells, such as pluripotent cells, that express C1-INH.

Described are nucleic acid regulatory elements that are able to enhance liver-specific expression of genes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. These are particularly useful for applications using gene therapy.