Using CRISPR/Cas9 to Edit Disease Out of the Genome


This post was originally written in 2014 by Kendall Morgan and updated in 2022 by Lucie Wilson. Lucie is an Addgene co-op from Northeastern University. 

There can be no doubt that CRISPR/Cas9 technology has been a breakthrough for the genome-editing field. It has the possibility to treat human genetic diseases, and several treatments are currently being tested in clinical trials. It is speeding the process of the discovery/development of genetic targets and RNA therapies. [6]. In 2014, we wrote a blog post on CRISPR’s potential for monogenetic diseases, the bulk of which you can find below.

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DAP arrays for multiplex gene editing


The natural CRISPR locus of a bacteria host encodes multiple guide RNAs (gRNAs) on a single array to target the genome of the invading phage pathogen. Over the past decade, CRISPR tools have leveraged such host-defense mechanisms to enable multiplex gene editing in a variety of cells and organisms. However, lengthy genetic payloads and insufficient transcription on the array have limited the scalability and efficiency of multiplex gene editing. Moreover, existing multiplex strategies have been facing difficulties in pairing with base editing and prime editing approaches. Recently, Xue Sherry Gao’s lab at Rice University has developed DAP arrays for efficient multiplex base editing (MBE) and multiplex prime editing (MPE) with only minimal payloads.

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Bioinformatics at Addgene


From the beginning, one of Addgene’s goals was to ensure the scientific community obtained high-quality data for our DNA-based reagents. Not only do we extract as much information as possible from depositing laboratories and associated publications, but our quality control (QC) standards have evolved over the years to keep up with the latest tools. In Addgene’s early days, we would ‘spot-check’ critical regions of plasmids deposited with us via Sanger sequencing, while more recently we have moved to full plasmid sequencing at our facility using Illumina MiSeq Next Generation Sequencing (NGS) technology.

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Summer SciComm Series: Master of SciComm


I didn’t truly address the question of what I wanted to do when I grew up until I was all but finished my undergraduate degree (and I hope I’m not alone in this sentiment). Entering the final semester of a neuroscience major, I was struck with the realisation that I didn’t want a career in neuroscience—whatever that entails. Despite being engrossed by the theory and findings of brain science, my curiosity did not ultimately translate to a desire to do the research itself. Several years of exclusively writing lab reports and empirical essays had also stirred some of my more creative faculties into motion, such that a desire to once again write for fun eventually saw an English minor tacked onto my BSc. With this decision I had unwittingly begun my journey towards a career in science communication, an established field of inquiry with which I was not yet familiar. The idea that my piecemeal degree might one day foster a meaningful day job only came into focus when an English tutor noted my eclectic mix of science and arts papers: “There’s a department at the university I think you should check out.”

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Gram Negative and Positive Bacteria


In a lab, you may have heard the phrase gram negative or positive being used to describe a species of bacteria, but what does it actually mean? What relevance does it have on the structure of a bacteria species and how it can be used in a lab? Here we’ll be talking about the ins and outs of this classification system, including its history, biology, and usages in the lab.

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Corralling and Cornering the Goldilocks Antibody


Have you ever been stuck googling about an antibody that will be essential to your new research project, but unsure which one will mark your target the best, or even which one will work for your application? Like many scientists, I have spent hours sifting through comments and papers to find the most reliable (and cost-effective) antibody for my experiment – the “goldilocks antibody.”

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Summer SciComm: Preprints


As a scientist, perhaps the most important part of your research is communicating the results of the work. Yes, those control experiments are important – but without letting other scientists know the results, your work won’t have any impact!

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A PhD in Science Communication


I’ve always been fascinated by the human body and how it works. I used to stay up past my bedtime, poring over my grandparents’ medical textbooks by torchlight under the covers. In high school, I went to all the optional sexual health sessions and reported the intel back to my shyer classmates. For my undergraduate degree, I majored in human anatomy, but I also took classes in biochemistry, microbiology, epidemiology, and bioanthropology. Studying human health science made me happy, but it wasn’t long before I started becoming disillusioned with the gulf between what I was doing in the lab and the interactions I was having outside of the “ivory tower.”

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