Patent classifications
C12N2740/15021
COMPOSITIONS AND METHODS FOR TREATING PHENYLKETONURIA
A lentiviral vector system for expressing a lentiviral particle is disclosed. The lentiviral vector system includes a therapeutic vector. The therapeutic vector comprises a phenylalanine hydroxylase (PAH) sequence for expressing at least one of PAH or a variant thereof, wherein the PAH sequence is truncated.
PRE-IMMUNIZATION AND IMMUNOTHERAPY
The present invention relates generally to immunization and immunotherapy for the treatment or prevention of HIV. In particular, the methods include in vivo and/or ex vivo enrichment of HIV-specific CD4+ T cells.
HIV pre-immunization and immunotherapy
The present invention relates generally to immunization and immunotherapy for the treatment or prevention of HIV. In particular, the methods include in vivo and/or ex vivo enrichment of HIV-specific CD4+ T cells.
HIV pre-immunization and immunotherapy
The present invention relates generally to immunization and immunotherapy for the treatment or prevention of HIV. In particular, the methods include in vivo and/or ex vivo enrichment of HIV-specific CD4+ T cells.
PRE-IMMUNIZATION AND IMMUNOTHERAPY
The present invention relates generally to immunization and immunotherapy for the treatment or prevention of HIV. In particular, the methods include in vivo and/or ex vivo enrichment of HIV-specific CD4+ T cells.
Method for NK cell transduction
The present invention discloses an in-vitro method for transferring biological material into activated NK cells with a pseudotyped retroviral vector particle or a virus-like particle thereof, comprising the steps a) activation of NK cells, and b) addition of said pseudotyped retroviral vector particle or virus-like particle thereof to said activated NK cells, wherein said pseudotyped retroviral vector particle or virus-like particle thereof comprises a modified baboon endogenous retrovirus (BaEV) envelope glycoprotein that is able of binding to and fusing with a hematopoietic cell membrane, thereby transferring biological material into said activated NK cells. Preferentially, the activating of NK cells is performed by the addition of a IL-1 family cytokine to the NK cells.
HIV PRE-IMMUNIZATION AND IMMUNOTHERAPY
The present invention relates generally to immunization and immunotherapy for the treatment or prevention of HIV. In particular, the methods include in vivo and/or ex vivo enrichment of HIV-specific CD4+ T cells.
HIV PRE-IMMUNIZATION AND IMMUNOTHERAPY
The present invention relates generally to immunization and immunotherapy for the treatment or prevention of HIV. In particular, the methods include in vivo and/or ex vivo enrichment of HIV-specific CD4+ T cells.
MYELOID-SPECIFIC PROMOTER AND USE THEREOF
Provided are a myeloid-specific promoter and a use thereof. The myeloid-specific promoter includes a nucleic acid sequence as shown in SEQ ID NO: 1 or SEQ ID NO: 2. The myeloid-specific promoter shows specificity to myeloid tissues. It initiates a gene expression with high efficiency in myeloid cells, but with relative low efficiency in non-myeloid cells. As such, the myeloid-specific promoter regulates the specific expression of a gene in myeloid tissues. The myeloid-specific promoter and the CYBB gene are inserted into a lentiviral vector, and the constructed lentiviral expression vector shows specificity to myeloid tissues and can effectively restore the expression of gp91-phox protein and restore the generation function of ROS, which is of great significance for CGD treatment.
Method for large-scale preparation of purified preparation of recombinant lentiviral vector at GMP grade
Provided is a method for large-scale preparation of a purified preparation of a recombinant lentiviral vector at the GMP grade. The method comprises: (a) providing raw material feed liquid to be purified that comprises recombinant viral vectors; (b) carrying out a microfiltration treatment on the feed liquid to obtain a microfiltered filtrate comprising the recombinant viral vectors; (c) optionally concentrating the filtrate to obtain a concentrated filtrate; (d) purifying the filtrate obtained in the previous step by means of chromatography to obtain a crude pure product comprising the recombinant viral vectors; and (e) subjecting the crude pure product obtained in the previous step to liquid exchange and elaborate purification to obtain the purified recombinant viral vectors.