Therapeutic genome editing requires effective and targeted delivery methods. The delivery of Cas9 mRNA using adeno-associated viruses has led to potent in vivo therapeutic efficacy, but can cause sustained Cas9 expression, anti-Cas9 immune responses and
In order to achieve long-term permanent levels of siRNA in the cell, stable transcription of siRNA can be achieved by viral siRNA constructs integrated into genomic DNA of target cells. From a practical standpoint, lentiviral vectors are an optimal delivery system for stable and effective (up to...
This study aims to use Vpx to target SAMHD1 in AML cells to improve ara-C sensitivity.In order to manipulate SAMDH1 levels using Vpx, different Vpx delivery systems were developed. These are virus-like particles (VLPs) packaged with different homologs of Vpx from Simian Immunodeficiency Viruses...
Use of the BLOCK-iT™ HiPerform™ Lentiviral Pol II miR RNAi Expression System with EmGFP to facilitate lentiviral-based delivery of miR RNAi to mammalian cells provides the following advantages: The BLOCK-iT™ Pol II miR RNAi Exp...
Cell membrane-based biovesicles (BVs) are important candidate drug delivery vehicles and comprise extracellular vesicles, virus-like particles, and lentiviral vectors. Here, we introduce a non-enzymatic assembly of purified BVs, supercharged proteins, an
These cells might be target cells for the delivery of thdd or ddth cells for the treatment of Parkinson's disease. This Example describes the construction of an HIV based vector which will deliver and express thdd or ddth genes, for example. [0076] The structure of a general HIV LLD ...
For constitutive expression of shRNA molecules in mammalian cells, use the BLOCK-iT™ U6 RNAi Entry Vector Kit or the BLOCK-iT™ Lentiviral RNAi Expression System. Other BLOCK-iT™ RNAi products are available to facilitate production and delivery of synthetic short interfering RNA (siRNA), ...
Use of the BLOCK-iT™ HiPerform™ Lentiviral Pol II miR RNAi Expression System with EmGFP to facilitate lentiviral-based delivery of miR RNAi to mammalian cells provides the following advantages: The BLOCK-iT™ Pol II miR RNAi Exp...
Lentiviral vectors are useful experimental tools for stable gene delivery and have been used to treat human inherited genetic disorders and hematologic malignancies with promising results. Because some of the lentiviral vector components are cytotoxic, t
Thus, high volumes of virus are needed when using the vector for gene delivery. The reason for this is the body's own immune system more readily attacks these vectors.Recently, attention has focused on lentiviral vectors such as those based upon the primate lentiviruses, e.g., human ...