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.
The team at Aldevron is excited to virtually celebrate our recent expansion in Madison and share the great progress we’re making and opportunities ahead in the world of gene therapy.
When we talk about the use of CRISPR-Cas9 technology, it’s usually in the context of developing treatments for human diseases. But there’s another aspect to the technology that has potential to have just as much impact on our lives: its use in the genetic modification of food crops.
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.
By Mark Osborn, Ph.D., Minnesota Stem Cell Institute, University of Minnesota
CRISPR/Cas9 is a vital part of our research at the University of Minnesota and the Cas9 recombinant protein, used at high concentration, has allowed for highly efficient modification of T-cells.
By introducing a Cas9 nuclease guide RNA complex (RNP), we target a specific spot in the genome, where the nuclease cuts the DNA. The DNA break is repaired in one of two ways: homologous recombination, which is high-fidelity, or non-homologous endjoining (NHEJ), which is more error-prone.
"Biomanufacturing" and "efficiency" are two terms that are not commonly seen together, but researchers at the University of Wisconsin-Madison Engineering Department along with protein manufacturing experts here at Aldevron are taking big steps to change that.
The application of Quick Response Manufacturing (QRM) theory to biological processes is a unique initiative whose goal of reducing lead times for manufacturing operations is the core focus of the UW and Aldevron partnership.
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.