Aldevron Breakthrough Blog: Protein

Contributing to a Cure for Sickle Cell Disease

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.

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Improving CRISPR Genome Editing to Treat Genetic Disease

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.

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Growing Agricultural Productivity with CRISPR-Cas9

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. 

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Efficient, reproducible results with Cas9

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.

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Cost-effective Cas9 – Higher concentrations bring results

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.

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