In recent days, there is high rise of diseases related to blood disorder like sickle cell anemia, thalassemia etc. where the red blood cells become distorted, rigid and adhesive. Sometimes they become fatal. Such diseases are the outcome of genetic mutation in a particular type of stem cell. CRISPR-Cas9 genome editing is a great solution for rectifying the mutation in the stem cells in lab. Then this edited cells can be easily placed the bodies of the affected people. The whole process can help in curing diseases. This makes the molecular biologists and biomedicine researchers to work more on this process.
There is a lack of protocol for targeting hematopoietic cells for gene targeting which influenced Rasmus O Bak et al, to develop a protocol for the invention, enhancement, in vivo and in vitro scrutinization of genome editing in hematopoietic stem cells (HSC). The study has been published in Nature Protocols in January 2018. The protocol involves analyses by combination of CRISPR/Cas9 process with utilization of rAAV6 and flow cytometry that will take 3 weeks for completion for in vitro HSC and 16 weeks for in vivo HSC. The protocol will help the researchers for making single nucleotide alteration in the genome and also gene editing at any specific place. Moreover, it will assist in discovering gene function relationship as well as various cell therapies.
