Nanotechnology in the Treatment of Sickle Cell Disease

Sickle Cell Disease (SCD) is a genetically inherited debilitating illness, caused by a point mutation (most commonly haemoglobin variant Hb S) in the beta-globin gene that requires early diagnosis after birth and constant monitoring throughout the life-span of the patient. Sickle cell anemia (SCA) is a condition that alters the morphology (size and shape) of RBCs. The disease phenotype is categorized into two most prominent phenotypes i.e. vaso-occlusive crisis and haemolysis. Both the phenotypes are the excruciating factors displaying debilitating symptoms in patients with SCA.
Although many compounds like hydroxyl urea, epigenetic agents with potential implications in fetal hemoglobin (Hb F) synthesis, anti-inflammatory agents, anti oxidants targeting cellular adhesion behaviour, however till now it’s of tremendous requirement to develop potential alternative compounds to target molecular cross talk rendering the pathophysiology of SCA. Currently, nano medicine enables highly specific medical treatment at the moleculer scale for curing disease and/or repairing tissue, The most promising enzymes for nano medicine applications are engineered nucleases, including zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and the clustered regularly interspaced short palindromic repeats (CRISPR) systems as powerful tools for genome editing through specifically cleaving genomic sequences. Using these materials, various types of nano particles are synthesized (gold nano particles, magnetic nano particles, etc.) are used in diagnosis likeimaging and treatment. Recently, Nanocar came into existence, which is acts as vehicle for bringing back the morphology of RBC, it contains four ferric wheels, two staffanes, graphitic sheets and buckytube, a reinforcing element with a rigid chassis and four alkyne axles that spin freely and swivel independently of one another. Palladium acts as a catalyst in attaching bucky ball (Buckminsterfullerene- 60-carbon alkenes) to the rest of the nanocar. Significant advancement in the field of nano medicine not only offers a major breakthrough in understanding the clinical severity associated with SCA but also provides a promising approach to target different pathways involving in pathophysiology. The present chapter will put emphasis on the potential role of nanotechnology in understanding and targeting the crucial pathophysiology of sickle cell disease.