CRISPR Gene Editing Technology

CRISPR Gene Editing Technology – “CRISPR” (pronounced “crisper”) stands for Clustered Regularly Interspaced Short Palindromic Repeats, which are the hallmark of a bacterial defense system that forms the basis for CRISPR-Cas9 genome editing technology. In the field of genome engineering, the term “CRISPR” or “CRISPR-Cas9” is often used loosely to refer to the various CRISPR-Cas9 and -CPF1, (and other) systems that can be programmed to target specific stretches of genetic code and to edit DNA at precise locations, as well as for other purposes, such as for new diagnostic tools.

CRISPR is a simple but powerful gene-editing technology that can modify, delete or correct disease-causing abnormalities at their genetic sources.

With the CRISPR gene editing systems, specialists can permanently modify genes in living cells and organisms and, in the future, may make it possible to correct mutations at precise locations in the human genome in order to treat genetic causes of disease. This corrective ability would be made possible by the gene-editing CRISPR technology. Other systems are now available, such as CRISPR-Cas13’s, that target RNA provide alternate avenues for use, and with unique characteristics that have been leveraged for sensitive diagnostic tools, such as SHERLOCK.

There are more than 10,000 known single-gene (or monogenic) diseases, occurring in about 1 out of every 100 births¹. Scientists and clinicians are now conducting pioneering research using CRISPR/Cas9 to address both recessive and dominant genetic defects, opening up the potential of gene editing to provide novel transformative gene-based medicines for patients with a large number of both rare and common diseases.

¹ Genomic Resource Centre: Genes and human disease, World Health Organization. http://www.who.int/genomics/public/geneticdiseases/en/index2.html