Genome Editing

Scientists have used a programmable CRISPR-based method called pgSIT to generate sterile but competitive males of the species Drosophila suzukii that poses a threat to food crops in western countries. If deployed at scale in the wild, the technology could potentially be effective in specifically and safely curbing this pest population. The authors claim that the method can be modified to target other pest species, precluding the need for environmentally unfriendly pesticides.

Molecular Assemblies’ enzymatic approach is based on how nature makes DNA—in water—to overcome the length, purity, and accuracy limitations of the chemical DNA synthesis method. The technology by Molecular Assemblies employs a template-independent DNA polymerase that can synthesize much longer DNA sequences with fewer errors. This two-step proprietary process uses non-toxic aqueous reagents, requires minimal post-synthesis processing, and can scale to longer DNA sequences.

Bluebird Bio’s Zynteglo has received FDA approval as the first gene therapy for people with beta-thalassemia who require regular red blood cell transfusions. The one-time gene therapy is custom-designed to treat the underlying genetic cause of beta thalassemia. Zynteglo, the company says, offers potentially curative benefit across ages and genotypes, through the achievement of normal (or near normal) total hemoglobin levels.

Alcyone Therapeutics assessed the benefits of several techniques for manufacturing AAV for gene therapy, including using baculovirus in insect cells, and a packaged cell line where information required to produce AAV is pre-written into the chromosomes of the cells. Having considered the likely difficulties of each approach, the company opted for transient transfection of mammalian cells using two or three plasmids.

In TODAY's GEN webinar, sponsored by WuXi, our expert panelists will discuss how they aim to develop technologies to improve Lentiviral vector yields and scalability. Specifically, we will hear about the challenges involved with the large-scale manufacture of Lentiviral vectors and how OXGENE’s LentiVEX™ plasmids are designed for high vector yields and can be used within transient manufacturing platforms.

High-fidelity Cas13 variants that target and degrade RNA have been developed that markedly reduce collateral effects. Through the development of a rapid and sensitive dual-fluorescence reporter system that detects off target cleavage, researchers were able to engineer and test over 200 engineered variants. In doing so, they found several Cas13 variants (including Cas13d and Cas13X) that exhibit efficient on-target activity but reduced collateral activity.

A genetic factor that leads to the absence of the protein EPS8 coincides with improper development of sensory hair cells in the inner ear. These cells normally have long hair-like structures, called stereocilia, that transduce sound into electrical signals that can be perceived by the brain. In the absence of EPS8, the stereocilia are too short to function, leading to deafness.

Cemvita Factory is on a mission to combat climate change using cost-effective, low-carbon solutions that have a net positive impact on the environment. The Houston-based biotech firm is a synthetic biology platform company whose central goal is to decarbonize the heavy industry, reducing the environmental impact of numerous industries from mining to oil and gas.

The deaths are the first fatal cases of acute liver failure that have been linked to Zolgensma, a gene therapy developed by AveXis. Novartis acquired AveXis for $8.7 billion in a deal completed in 2018. A year later in May 2019, the FDA approved Zolgensma for the treatment of spinal muscular atrophy (SMA) in pediatric patients less than two years of age with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Scientists have developed a toolbox of transcriptional regulators for plants and show that they can be used to construct synthetic genetic circuits that predictably modify root growth. Their work is an important step in designing crops with improved productivity that adapt well to climate change.