Stem Cells Applications in Therapeutics and Site-Specific Genome Editing Through CRISPR Cas9 System

Author(s): Muhammad Javed Iqbal, Muhammad Mukheed, Alisha Khan, Saba Irfan, Usama Hayat, Muhammad Umair Khalid, Muhammad Ali Raza, Kainat Amjad, Marriyam Tallat

Stem cells are immature cells that have ability to differentiate into all specific and mature cells in body. The two main characteristics of stem cells are self-renewable and ability to differentiate into all mature, functional, and adult cell types. There are the two major classes a) pluripotent stem cells which have potential to differentiate in all adult cell and b) multipotent stem cells which have capacity to differentiate into many adult cells but not in all cell types. Due to the self-renewable ability stem cells are used in therapeutics, tissue regeneration, disease modeling, regenerative medicines and to treat cardiovascular diseases, neural disorders such as Parkinson’s disease and most importantly to treat carcinomas. The human induced pluripotent stem cells provide a great platform to study and treatment of human diseases because these can differentiate into many functional and specialized adult cells of body. The genome editing tools such as CRISPR Cas9 system and TALENs are used to generate multiple DNA variants in hPSCs by inducing site specific mutations, frame shift mutation and deletion. In present days CRISPR Cas9 is more efficient and frequent method for genome editing which is derived from bacterial cell.

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