Gene editing is a technique that allows scientists to alter the DNA of living organisms. The most common tool to introduce these alterations is the clustered regularly interspaced short palindromic repeats (CRISPR). CRISPR was originally extracted from bacteria, where it helps protect from foreign invaders, such as viruses. This tool was later used to alter genes in mammalian cells with significant promise.
Another way to regulate genes is using RNA interference. This technique uses small RNA segments to interfere with the expression of specific genes. This can be used to turn off genes or reduce the amount of proteins expressed by them. RNA interference has been used to study the function of different genes, and to treat diseases like cancer and hepatitis C.
The discovery of CRISPR
CRISPR is part of the adaptive immune system in the archaea and bacteria where it is responsible for defending against foreign nucleic acids, introduced via plasmids and phages (1). Even though the acronym CRISPR was introduced in 2002 (2, 3), the immunologic rules of these repeated DNA sequences were discovered in 2005 by Mojica and colleagues (4). Following that, Charpentier and Doudna reported the mechanism and biomedical characteristics of the CRISPR-associated protein 9 (Cas9) (5). However, the most significant breakthrough was in 2015 when Zhang was successful in adopting the CRISPR-Cas9 in eukaryotic cells for targeted genome editing (6). The major benchmarks of the development of CRISPR systems are summarised here (7).
The inner workings
The potential of CRISPR
The rise of CRISPR has revolutionised gene editing, which holds vast potential for treating genetic diseases. CRISPR is a simple, efficient, and precise tool for modifying genes. It has already been used to treat a number of diseases in humans and animals. The ability to precisely edit genes has the potential to cure many genetic diseases, including cystic fibrosis, sickle cell anemia, and muscular dystrophy. CRISPR may also be used to treat cancer by disabling the genes that allow tumours to grow, among other diseases that originate from genetic mutations.
We offer a range of services for the identification of target genes and the development of specialised approaches to edit and regulate them. Whether you are looking for new targets to edit or need help developing an approach to achieve specific results, we are here to help achieve your desired outcome.
The second step in the process development is selecting and designing the right tools and components of your therapeutics. We will determine the right CRISPR variants from our extensive toolbox, select the most efficient gRNA sequences, design novel siRNA constructs using cutting-edge technologies, or map the right pDNA for cell line development.
Testing is an essential part of the development journey. We work with you to either develop a new cell line or test the designed platform on specific cell lines, depending on your needs. We will then select the top candidate components for the final validation.
We use several approaches for validating the success of the designed platform. Depending on the design, we will validate the outcomes with sequencing, profiling mRNA expression, or testing for protein expression. This will help us to confirm that the platform is working as intended and producing the desired results.
Prorenata Biotech is specialised in drug delivery for genetic medicine. We design and develop scalable Lipid Nanoparticle (LNPs) solutions that are tailored to specifically meet your requirements. Our LNPs are designed from the early preclinical stages, all the way to GMP-grade LNPs for clinical applications.