Patent classifications
C12N15/86
Adeno-Associated Viral (AAV) Vectors for Tissue-Targeted Expression of Therapeutic Genes
Described herein are compositions and methods for tissue-targeted expression of therapeutic genes, using AAV expression vectors that reduce the risk of toxicity associated with AAV gene therapy in the CNS by de-targeting the vulnerable neurons cells including the DRG cells on gene expression, and de-targeting the liver, a major suspect for over-expression in the periphery.
METHOD FOR MANUFACTURING INDUCED PLURIPOTENT STEM CELLS
According to the present disclosure, there is provided a method for manufacturing pluripotent stem cells including introducing a reprogramming factor into cells and seeding the cells, into which the reprogramming factor is introduced, at a low concentration and culturing the cells.
METHOD FOR MANUFACTURING INDUCED PLURIPOTENT STEM CELLS
According to the present disclosure, there is provided a method for manufacturing pluripotent stem cells including introducing a reprogramming factor into cells and seeding the cells, into which the reprogramming factor is introduced, at a low concentration and culturing the cells.
ANELLOSOMES FOR DELIVERING SECRETED THERAPEUTIC MODALITIES
This invention relates generally to anellosomes and compositions and uses thereof.
ANELLOSOMES FOR DELIVERING SECRETED THERAPEUTIC MODALITIES
This invention relates generally to anellosomes and compositions and uses thereof.
ALTERING TISSUE TROPISM OF ADENO-ASSOCIATED VIRUSES
This disclosure provides compositions and methods for altering or changing the tissue tropism, e.g., liver tropism, of adeno-associated viruses (AAV).
ALTERING TISSUE TROPISM OF ADENO-ASSOCIATED VIRUSES
This disclosure provides compositions and methods for altering or changing the tissue tropism, e.g., liver tropism, of adeno-associated viruses (AAV).
ENGINEERED IMMUNE CELLS
The invention relates to an immune cell that is capable of antibody-dependent cellular cytotoxicity and which comprises a nucleic acid sequence encoding a secreted antigen binding protein. The invention also concerns a method of producing the immune cell and medical uses for the immune cell.
ENGINEERED IMMUNE CELLS
The invention relates to an immune cell that is capable of antibody-dependent cellular cytotoxicity and which comprises a nucleic acid sequence encoding a secreted antigen binding protein. The invention also concerns a method of producing the immune cell and medical uses for the immune cell.
REGULATABLE EXPRESSION SYSTEMS
Provided herein are regulatable expression systems and methods of using said regulatable expression systems to express proteins of interest. The regulatable expression systems comprise a unidirectional regulatable promoter operably linked to a single transcription unit encoding a protein of interest, a ribosome skip, and a transactivator protein.