Every year, an estimated 300,000 children are born globally with a severe form of sickle cell disease. This is a genetic disease that causes red blood cells to be a sickle shape, leading to episodes of pain and anaemia, along with the potential of stroke or kidney damage.
CRISPR genome editing has enormous potential as a means to treat genetic disorders, such as sickle cell disease. However, despite massive success in pre-clinical experiments, the impact of CRISPR in the clinic has been relatively limited.
One problem is a lack of options for large-scale delivery of CRISPR enzymes to patients.
Earlier this month, Aldevron and Synthego co-sponsored a GEN webinar exploring excitingnew research into how to deliver genome editing enzymes in vivo into patient cells.
By Krishanu Saha, Ph.D., Department of Biomedical Engineering & Wisconsin Institute for Discovery, University of Wisconsin-Madison
At the University of Wisconsin - Madison, one focus of our group is understanding and optimizing CRISPR-Cas9 gene editing for therapeutic and disease modeling applications. To conduct our research, we need reliable, consistent and highly efficient Cas9 protein.
Model system for Cas9 gene editing
To perform targeted gene editing, the Cas9 protein, which cuts the genome, and a guide RNA (gRNA) that encodes where in the genome to cut, need to be complexed together into a ribonuclear protein (RNP) complex and transfected to cells to reach the nucleus. Once the DNA is cut, imprecise DNA repair may cause disruption at the cut site, which can result in knock out of a gene.
Groundbreaking technologies arise every decade or so, advancing genomics research and the development of new medicines. Restriction enzymes in the 70s, polymerase chain reaction in the 80s and next-generation sequencing in the 90s all represented huge leaps forward. Recently, however, the CRISPR revolution has shown the potential to overshadow them all.
CRISPR, or clustered regularly interspaced short palindromic repeats, is a long-sought breakthrough for making highly specific changes to genetic codes. Compared to established technologies such as zinc-finger nucleases (ZFNs) and transcription activator like effector nucleases (TALENs), the CRISPR system using Cas9 nuclease is faster, easier, more precise and more flexible.
CRISPR technology will have great impact on basic and applied life-science research. Therefore, important questions should be asked when choosing a partner that can develop and manufacture CRISPR components such as Cas9 to meet your present and future needs for Cas9 product design, scale and quality.
We’ve reviewed three important reasons why experience and expertise matters when choosing such a partner.