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Genome Editing

At Orchard, Moving from Pruning to Planting

CEO Bobby Gasoar, MD, PhD, made his presence felt at Orchard Therapeutics in May, leading the company in carrying out a strategic plan that included eliminating about 25% of its workforce, or 60 jobs, and narrowing its near-term R&D pipeline programs to four.

An Efficient Single-Nucleotide-Editing CRISPR

Scientists succeeded in producing the first transgenic mice using a base editor made of cytidine deaminase fused to CRISPR/Cas9

Solid Recommits to DMD Gene Therapy on Clinical Hold, as Stock Plunge Sparks Lawsuits

Solid Biosciences restated its commitment to developing its lead candidate, the Duchenne muscular dystrophy (DMD) gene therapy SGT-001, despite the FDA’s full and partial...

A CRISPR Vision for the Future of Cell Therapy

In this webinar from GEN and The CRISPR Journal, T.J. Cradick, PhD, —who was until recently head of genome editing at CRISPR Therapeutics—will introduce how genome editing is used to engineer cell therapies.

UniQure Says It Will Not Pursue EC Marketing Renewal for Glybera Gene Therapy

Firm cites low demand and high costs associated with maintaining manufacturing infrastructure and regulatory-related activities as reasons for withdrawing the gene therapy from the European market
Nile Tilapia

A Fish Called CRISPR: Blocking Melanin to Boost Commercial Demand for Tilapia

Scientists at Israel’s Agricultural Research Organization (ARO), Volcani Center in Rishon Lezion, have used CRISPR-Cas9 genome editing tools to introduce indel mutations in a membrane transport protein, slc45a2, to create a new stable strain of tilapia that does not develop suspicious red and black blotches on their skin and instead acquires appealing, uniform, red skins that drive up their market price. These findings demonstrate genome editing is useful for the generation of improved and traceable phenocopies of commercially popular and profitable traits that do not require large-scale genetic manipulations that generate new phenotypes but subtle genetic changes.

New Method Enables Multiplex Genome Engineering

A consortium of scientists working at MIT, Harvard, Tsinghua University, Columbia University, and the Rockefeller University announced in the January 3 online edition of Science that they had devised a way to enable simultaneous editing of several sites within the mammalian genome.

Gene Therapy for Sickle Cell Aces Vital Preclinical Test

Decades-old discovery about fetal hemoglobin on track for clinical trial in coming year.
The structure of the BurrH binding domain

Look Out CRISPR, BuD-Derived Gene Editing Tools May Be Gaining On You

Researchers detail the binding domain of BurrH, a DNA-binding protein. They also reprogrammed the domain, called BuD, showing its potential as a gene-editing tool.
The crystal structure of SpyCas9 features a nuclease domain lobe (red) and an alpha-helical lobe (gray) each with a nucleic acid binding cleft that becomes functionalized when Cas9 binds to guide RNA. [Lawrence Berkeley National Laboratory]

Seeing How Cas9 Got Its Groove Back, in 3D

High-res, 3D imaging of Cas 9 reveals RNA-mediated conformational activation.