Highlights • Apo DdmD assembles as an autoinhibited dimer for plasmid nicking • DdmE is a DNA-guided, DNA-targeting Argonaute-like protein and prefers short guides •
Abstract To maximize the impact of precision medicine approaches, it is critical to identify genetic variants underlying disease and to accurately quantify their functional effects.
Editing genes in human embryos could one day prevent some serious genetic disorders from being passed down from parents to their children — but, for
Highlights • 664 patients and 154 CFTR mutations represented in an organoid biobank • Adenine base editors enable efficient repair of nonsense mutations in CFTR
his story introduces Science’s CRISPR in China series, supported by the Pulitzer Center. For many people, CRISPR plus China equals the biophysicist He Jiankui, who
In 2018, scientists rode the activist momentum of 2017 to step out of the lab and into the public sphere: There was an unprecedented surge
Newly identified anti-CRISPR proteins block Cas12a, a cousin of the Cas9 nuclease, both of which are widely used in genome editing, two teams of researchers
Innovation comes in many forms, molded into various outlooks, adapted to shifting time frames. Sometimes, technological and conceptual progress is undergirded by a more expansive
Researchers have edited the human genome before, but always in cells outside the body. Now, biotech company Sangamo Therapeutics is recruiting participants for clinical trials
In a first, a man has received a therapy aimed at editing the genes inside his body. The Associated Press reports that 44-year-old Brian Madeux,
Base editing is a relatively recent genome-editing technique that swaps one DNA base pair for another, offering researchers the potential to correct harmful, single-nucleotide mutations
Fusing an RNA-editing enzyme to an RNA-targeting Cas protein has enabled researchers to edit specific nucleotides within RNA molecules in human cells. The approach, called
CRISPR, the wildly popular genome-editing research tool, was invented in the West, but it is speeding toward potential human applications in China. Last week, the
A team of Chinese researchers who were the first to report having applied CRISPR to human embryos have done it again—this time upgrading their technique
Scientists have used the gene-editing CRISPR-Cas9 system to suppress the gene encoding the protein OCT4 in experiments with human embryos. According to the results, published
Last week, word got out that a team of scientists had edited viable human embryos for the first time in the United States. Details of
A minor proportion of people harbor a homozygous mutation in CCR5—a gene that encodes a receptor found on immune cells—that thwarts HIV’s attempts to get
Researchers have edited the human genome before, but always in cells outside the body. Now, biotech company Sangamo Therapeutics is recruiting participants for clinical trials
Microbes use a number of CRISPR-Cas systems for immunity, but it’s the class 2 system, typically utilizing the nuclease Cas9, that has been adapted for
Targeted genome editing via engineered nucleases is an exciting area of biomedical research and holds potential for clinical applications. Despite rapid advances in the field,
Like other retroviruses, the genetic material of HIV wedges itself into the genome of its human host. While antiretroviral therapies are effective at repressing HIV,
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