Genome Editing

Applied Genetic Technologies Corp. (AGTC) plans to launch a pivotal trial of its gene therapy candidate for X-linked retinitis pigmentosa (XLRP) by the end of this year, following strong positive six-month data from its ongoing Phase I/II trial.

Nanoparticles built of poly(beta-amino ester)s, or PBAEs, have been used to deliver suicide gene therapy to tumors in mouse models of brain cancer, specifically, medulloblastoma and atypical teratoid/rhabdoid tumors. By preferentially targeting tumor cells over healthy cells and exerting robust killing power, the nanoparticles show potential for safe and effective nonviral gene therapies, which may be especially valuable against pediatric CNS malignancies.

Weill Cornell Medical College researchers show the feasibility of using gene therapy to treat the progressive neurodegenerative disorder chronic traumatic encephalopathy (CTE). The study demonstrated the effectiveness of direct delivery of gene therapy into the brain of a mouse model of CTE.

It’s January again, the month we say goodbye to one year and hello to another. If we chose to observe tradition, we would do the usual thing: present a look backward and a look forward. But that’s just two directions—not enough for us. We need to look in every direction at once because we cover multiple disciplines in the life sciences. If you want a 360° view, you should try gazing into a crystal ball. Such a tool of divination is to be found in our January issue’s special feature, “The Outlook for 2020 and Beyond.”

In a chat with GEN’s Kevin Davies, pioneering scientist George Church covers a lot of ground: genome recoding, multiplex editing, human-safe xenotransplants, germline editing, and (of course) mammoths.

Quiet confidence comes naturally to Bio-Techne, which supplies a staggering array of biotech products. The company boasts a 43-year history and offers more than 300,000 products. Bio-Techne has product lines that cover proteomic instrumentation, assays, antibodies, proteins, adoptive cellular therapy products, bead technology for cell aggregations, gene editing transposon technology, a liquid biopsy platform, an automated multiplex immunoassay platform, and quality control technology for the clinical market. Yet the company is anything but complacent. It’s working to establish itself in cell and gene therapies.

Cryo-electron microscopy images have been taken of INTEGRATE, a new gene editing tool in which CRISPR machinery and transposon machinery are linked and with which large genetic payloads may be inserted into the genome without introducing double-stranded breaks. The images have been used to generate an atomic resolution model that clarifies INTEGRATE’s mechanism of action, which includes the downstream recruitment of additional transposase proteins. The model could guide protein engineering efforts to improve the system’s programmable DNA insertion capabilities.

Thirteen months after He Jiankui shook the international scientific community with the news of an experiment to create the first gene-edited babies, a Chinese court has sentenced He and two colleagues to three years in prison and a fine of roughly $400,000. Producing the CRISPR babies, which did not gain the support of the scientific community, brought the ethics that surround CRISPR genome editing technology to the forefront of scientific and ethical debates.

One limitation of gene therapy is the lack of small genetic switches that would allow for the control of transgene expression. Now, a team of researchers has developed a reversible RNA on-switch that could be embedded into gene therapies to control dosing, offering what may be the first viable technique for adjusting the activity levels of therapeutic genes. These small, efficient RNA switches may improve the safety and efficacy of gene therapies.

Some suggest that contract manufacturers could help to identify common manufacturing challenges if customers are willing to work together and share information about non-competitive areas of production.