探索载体元件

The more we learn about the world around us, the more resources we have to make life better. From plasmids to CRISPR, we continue to take discoveries from the natural world and utilize them for further discoveries and treatments. Our "My Favorite Building Block" series takes the individual pieces of the castles we build in molecular biology and examines them in detail. Before we begin looking at the pieces in depth, let's take a moment to step back and set the stage by reviewing some basics in c...View more

基因治疗医学

Cargo size is key Duchenne Muscular Dystrophy (DMD) is a genetic disease that affects ~1 in every 5,000 people. The disease results in muscle weakness and loss, eventually resulting in death due to a genetic mutation in the dystrophin gene. Dystrophin is the largest known gene in the human genome and spans over 2.4 megabases of DNA, taking ~16 hours to be transcribed by RNA polymerase. Due to the large size of this gene, viral vector systems like AAV and lentivirus are not capable of delivering ...View more

探索载体元件

Plasmids are at the heart of gene delivery experiments, as they can be used to over-express a transgene, knock a gene out, or modulate gene expression. Often a single plasmid is the workhorse of the experiment. That is a lot of responsibility hanging on this tiny circle of DNA, so there are many important components to consider. Here we'll review some basics of plasmid components to make sure everyone is accounted for in your experimental design. Plasmid basics Our first post in "My Favorite Bu...View more

探索载体元件

CRISPR-Cas9 technology has revolutionized genome editing by providing efficient tools for gene knockouts, knockins, knock-ups, knock-downs, and more. In addition to being an invaluable tool in labs around the world, the first CRISPR therapy received FDA approval in December 2023 with many more therapies on the horizon. New components and approaches are constantly being developed to fully utilize this elegant system, so designing effective CRISPR experiments for research or clinical use can be dif...View more

灵光一闪时刻

When I was a graduate student, we called the lab's PCR machine the "Magic Box," because the reagents that we put inside could produce exactly what we were looking for, something completely unexpected, or nothing at all. If a reaction did not work, it was often difficult to tell exactly what needed to be changed. Was it the primers? The template? The shoes you were wearing that day? Understanding what is happening in that Magic Box can ensure that you are able to design successful PCR experiments...View more

聚焦载体科学

Adeno-associated virus (AAV) is a popular viral vector used for gene delivery and has rapidly overtaken other delivery systems as the vector of choice for in vivo applications and therapeutics development. This is largely due to its enhanced safety profile, owing to its lack of pathogenicity and low immunogenicity, as well as broad tissue tropism enabled by the diversity of naturally occurring serotypes. Throughout this article, we will discuss how AAV became so widely used, particularly for ther...View more

聚焦载体科学

AAV is emerging as a popular tool for gene delivery in the lab and in the clinic but deciding which serotype to use when first starting your experiment can seem like an overwhelming task. Although a broad base of literature exists to help guide your decision, certain scenarios exist where you must go beyond the published data. In this guide we will dive into what specifically differentiates one AAV serotype from another, how to best target your cells, and highlight a VectorBuilder product that ca...View more