Role of CRISPR-Cas/9 in Hematological Disorders: A Review

Over the past two decades, advances in molecular biology and genetics have greatly extended our knowledge of hematological diseases. Every year hematological disease are affecting million people from malignancies to acquired coaglupathiesis. However, the characterization of genes of interest remains a great challenge. The optimal way to unravel the roles of particular genes is to manipulate their functions via gene deletion, insertion or modification. Genome engineering is defined as the deliberate modification of an organism’s genetic material. From the 1980s several studies unravel the genetic mutation and their effect on human disease to determine the malignancies and advance diagnosis, prevention and diagnosis. Earlier gene editing technology were homologous recombination which was less efficient, time consuming & laborintensive process. Later in subsequent years, Zinc finger nucleases [ZFNs] & transcription activator-like effector nucleases [TALENs], was introduced to stimulate cellular DNA repair pathways. But there are so many limitations, so to overcome those limitation CRIPR associated CAS9 protein has introduce to start a new era of genome editing. From past few years CRISPR have change the path of genome editing be developing plant and animal models and the alleviation of human disease like hematological disorders. According to research crRNA sequence have significance role in targeting DNA [1,2]. To established CRISPR/Cas9 Jinek et al, 2012 introduce crRNA and tracrRNA into a single 100 nucleotide and a guider RNA which helps in the breaking of double stranded DNA and thus successfully genome editing done in human and mouse cells [3]. In the hematopoietic setting, CRISPR/Cas9 gene editing has been applied both in research and in clinical translation studies. CRISPER/Cas9 can be used to treat the clonal hematopoiesis by recapitulate the genetic mutation in patient. The main goal is to restore the immune system by the next generation genomics studies coupled with CRISPR/Cas9. Several companies develops their Abstract