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Adeno Associated Virus (AAV) for Cell and Gene Therapy

Posted by Guest Blogger on Nov 7, 2017 8:59:12 AM

This post was contributed by guest blogger Harshana S De Silva Feelixge.

Subscribe to Viral Vectors Blog Posts

Gene therapy technologies hold great promise for improving or potentially curing human diseases that were previously thought to be incurable. Rapid advances in next generation sequencing technologies have allowed scientists to quickly identify underlying genetic causes of some human conditions, opening up new avenues for therapeutics that treat disease at the molecular level. For instance, if a disease is caused by a mutation in a single gene, it can potentially be treated by correcting the mutation or replacing the gene. A notable example is the treatment of Severe Combined Immune Deficiency disease (SCID-XI), also known as bubble boy syndrome. This disease is caused by mutations in the common cytokine receptor gamma chain (c) and is characterized by a lack of immune cell development and function. To date, gene therapy has been used to treat 10 infants with this disease. To do so, their T-cells were grown in vitro, their mutations corrected, and the T-cells were transferred back into the infants. Almost all patients have achieved persistent immunological reconstitution with a normally functioning T cell repertoire (1).

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Topics: Viral Vectors

Cultivating Community Science at BosLab

Posted by Guest Blogger on Nov 2, 2017 9:35:33 AM

This guest post was contributed by Angela Kaczmarczyk, co-organizer at BosLab.

Biotechnology is no longer just for those working in academia or industry. At BosLab, we identify ourselves as part of a network of independent Do-it-Yourself (DIY) biology laboratories working towards enabling everyone to participate in the biotech revolution. Our lab exists in what appears to be a funky old garage, but looks can be deceiving. Step into this building and you will find a bike kitchen on the first floor and upstairs you will find our fully-equipped laboratory for citizen scientists. In addition to maintaining a laboratory to support both individual and community science projects, we offer classes, workshops, book clubs, seminars, and social events.

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Topics: Scientific Sharing

With an Eye Towards the Future, We Look Back at the March for Science

Posted by Guest Blogger on Oct 24, 2017 9:54:05 AM

This post was contributed by guest blogger Stephanie Hays, a researcher at the University of California Berkeley.

It’s been half a year since the march for science on April 22, 2017. While experiments (and editors) can move slowly, news about possible broad changes to policies seems to break everyday. Many researchers and supporters of science marched to advocate for scientist participation in government, evidence to inform policies, a protected place to work, funding for science, and numerous other reasons. It is important to remember that we, scientists and non-scientists alike, need to stay active and involved if we wish to make positive change. Below I present perspectives on the March for Science from researchers all over North America. I hope that these perspectives, the optimism they encapsulate, and the solutions they promote help motivate you to spend a little time advocating for science and getting yourself out there for the next six months and beyond.

Disclaimer: The views represented below are those of the writers and do not necessarily represent the views of Addgene.

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Topics: News, Science Communication

Advanced Uses of Cre-lox and Flp-FRT - A Neuroscientist’s View

Posted by Guest Blogger on Oct 19, 2017 9:54:49 AM

This post was contributed by guest blogger Katrin Michel.

Cre-lox is an incredibly popular and powerful site specific recombinase (SSR) system, but it only gives you a single level of control without modification - either Cre is there or it’s not. Cre-mediated possibilities for site specific (and often cell type specific) control of DNA recombination and gene expression can be advanced by the coordinated use of fellow SSR system FLP-FRT. In addition, a variety of means to spatiotemporally control FLP and Cre expression have been developed. Read on to learn more about FLP-FRT, Cre-lox, and how combinations of FLP and Cre enable additional levels of genetic control.

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Topics: Plasmid Technology, Cre-lox, Techniques

3D Printing Meets CRISPR Cas9

Posted by Guest Blogger on Oct 17, 2017 8:55:24 AM

This post was contributed by Tim Herman, director of the MSOE center for BioMolecular Modeling and the CEO of 3-D Molecular Designs.

Have you ever held your favorite protein in the palm of our hand? Well, actually – have you ever held a model of your favorite protein in your hand? At 3D Molecular Designs, we create physical models of proteins using 3D printing technology. The tagline for our company is “…where molecules become real”.  We sometimes forget that the models we create are around 30 million times larger than the real thing. Nevertheless, the models are compelling, and I encourage you to hold one.

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Topics: CRISPR

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