Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success
نویسندگان
چکیده
Hemophilia B is a serious hemostasis disorder due to mutations of the factor IX gene in the X chromosome. Gene therapy has gained momentum in recent years as a therapeutic option for hemophilia B. In hemophilia, reconstitution with a mere 1–2% of the clotting factor improves the quality of life, while 5–20% suffices to ameliorate the bleeding disorder. A paper by Guan et al (2016) in this issue of EMBO Molecular Medicine reports on the direct CRISPRs/Cas9-mediated correction in the liver of a hemophilia-causing point mutation in FIX.
منابع مشابه
Synthesis a New Viral Base Vector Carrying Single Guide RNA (sgRNA) and Green Florescent Protein (GFP)
CRISPR/Cas9 system is a powerful gene editing tool in vivo and in vitro. Currently, CRISPR/Cas9 delivery cells or tissue with different vehicles are available, and Adeno- associated virus (AAV) in one of them. Due to AAV packaging size limitation, AAV base vectors that carry CRISPR/Cas9 system do not have florescent tag like GFP for simple detection and navigation of cells, containing AAV. The ...
متن کاملThe new genomic editing system (CRISPR)
Over the past decades, progression in genetic element manipulation, and consequently, the treatment of diseases has been remarkable. It is worth noting that these genetic manipulations perform at different levels, including DNA and RNA. The earlier genomic editing techniques, including MN, ZFN , TALEN , performing their functions by creating double-stranded breaks (DSBs), and after breakage, th...
متن کاملCRISPR/Cas9, a new approach to successful knockdown of ABCB1/P-glycoprotein and reversal of chemosensitivity in human epithelial ovarian cancer cell line
Objective(s): Multidrug resistance (MDR) is a major obstacle in the successful chemotherapy of ovarian cancer. Inhibition of P-glycoprotein (P-gp), a member of ATP-binding cassette (ABC) transporters, is a well-known strategy to overcome MDR in cancer. The aim of this study was to investigate the efficiency and ability of CRISPR/Cas9 genome editing technology to knockdown ABCB1 gene expression ...
متن کاملDeveloping oncolytic Herpes simplex virus type 1 through UL39 knockout by CRISPR-Cas9
Objective(s): Oncolytic Herpes simplex virus type 1 (HSV-1) has emerged as a promising strategy for cancer therapy. However, development of novel oncolytic mutants has remained a major challenge owing to low efficiency of conventional genome editing methods. Recently, CRISPR-Cas9 has revolutionized genome editing.Materials and Methods: I...
متن کاملAdvancing Chimeric Antigen Receptor-Engineered T-Cell Immunotherapy Using Genome Editing Technologies: Challenges and Future Prospects
Chimeric antigen receptor engineered-T (CAR-T) cells also named as living drugs, have been recently known as a breakthrough technology and were applied as an adoptive immunotherapy against different types of cancer. They also attracted widespread interest because of the success of B-cell malignancy therapy achieved by anti-CD19 CAR-T cells. Current genetic toolbox enabled the synthesis of CARs ...
متن کامل