CRISPR-Cas9 gene editing technology in human gene therapy: the new realm of medicine

ABSTRACT

Gene therapy has a huge clinical relevance in the present therapeutic world and is one of the many research fields of biology which received many benefits from the recent advancements of modern clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing technology. Researchers are on the way to make significant changes in the ways of treating genetic abnormalities. An increase in the number of approved clinical trials of CRISPR based gene therapy shows we are not too far from eliminating deadly diseases such as acquired immunodeficiency syndrome (AIDS), cancer and many inherited genetic conditions from the society. However, there are some challenges associated with the development of CRISPR technology in medical field most of which revolves around its safety, efficiency and ethics. Lack of an optimized method by which the CRISPR-Cas9 expression cassette can be delivered to cells is one of the main challenges when it comes to its application in human gene therapy. Although viral vectors are the most common delivery systems used in gene therapy, recent researches show promising results on using lipid-based l delivery systems such as liposome-templated hydrogel nanoparticles (LHNPs). As these could eliminate the safety concerns of using viral vectors, it is expected to have potential therapeutic applications in future. Nevertheless, the efficiency of non-viral systems is still not fully comparable with that of viral vectors. Hence, CRISPR based therapies might take longer than expected tobe prevalent in the medical field. In this short review, the recent advances of CRISPR technology in gene therapy is discussed along with its challenges and limitations.