Tools & Techniques

How CRISPR stacks up against more established new modalities

The first indication that CRISPR-based gene editing can work when injected systemically brings competition from the emerging technology closer to reality for creators of RNAi and antisense therapies, but...

Flexible platform: how NCI’s Sharpless thinks COVID variants could boost personalized therapies

As regulators embrace platform technologies to rapidly update COVID-19 vaccines, they are laying the groundwork for personalized cancer and rare disease therapies, according to NCI Director Ned Sharpless....

Why Verve chose base editing over standard CRISPR for its lead CV candidate

Verve’s non-human primate data makes the case that base editing can produce durable and efficient changes in protein production by the liver, with a clear route to protection from heart attack. Six-month data shared by...

What DeepMind’s leap means for structure-based drug developers

The step change in protein folding prediction pioneered by Alphabet’s DeepMind stands to widen the funnel of targets tackled with structure-based platforms, including newly discovered proteins from...

Doudna and Charpentier, CRISPR pioneers behind a spate of gene editing companies, awarded Nobel Prize

Jennifer Doudna and Emmanuelle Charpentier have won the 2020 Nobel Prize in Chemistry eight years after their joint discovery that the CRISPR-Cas9 bacterial immune defense system can be harnessed...

Human germline editing report takes first steps toward concrete scientific guidelines

A report released Wednesday by an expert panel on human germline editing is the first to go beyond calls for caution and consensus and start to spell out technical definitions of safety.  Like every other...

Lurie winner and incoming Genentech R&D chief Aviv Regev looks to gene programs to untangle complex diseases

Single cell pioneer Aviv Regev thinks the gene networks revealed by the technology are on the verge of delivering new ways to attack disease biology. Her emphasis is on the idea that uncovering how genes...

A plasma tau marker gains support for diagnosing Alzheimer’s disease

Two studies published this week advance the search for blood tests for Alzheimer’s disease, but it’s not yet clear how transformative the plasma markers will be. Both studies came to the same conclusion: one form...

Data Bytes: the evolving CRISPR landscape

CRISPR pioneer Jennifer Doudna is adding a Cas protein to the toolbox with features that could make it a good gene editing tool. In a Science paper published on Thursday, the University of California Berkeley...

Precise base editing of mitochondrial DNA

David Liu and colleagues at the Broad Institute and University of Washington have achieved single-base editing of mitochondrial DNA for the first time, overcoming a limitation of CRISPR-based technologies and opening up new avenues for...

Tapping astrocytes to replace lost neurons in Parkinson’s

In the latest example of a regenerative medicine approach that involves transforming one cell type in the body into another, a University of California San Diego team has shown it can replace lost dopamine neurons...

AI tools against COVID-19: a view from where the rubber hits the road

Making machine learning an effective tool in the fight against COVID-19 requires broad access to data, scrupulous checks for bias, and strategies to balance speed and accuracy, according to researchers with AI-based diagnostics and literature-mining...

Spark’s approach to the problem of redosing AAV gene therapies

Spark aims to solve one of the big problems plaguing gene therapy -- immune responses against viral vectors -- by adapting a strategy Hansa has been developing for transplant rejection. Past infections with adenoviruses or...

Denali offers rare peek into the science behind its CNS delivery platform

Denali is lifting the veil on the delivery technology it’s using to transport large molecules into the CNS, detailing a differentiated strategy for hijacking the transferrin receptor. The company revealed features of its large molecule...

Octant emerges with $30M to untangle, exploit pathways hit by dirty drugs

With a $30 million series A round led by Andreessen Horowitz, Octant is using genetic bar codes to sort out the pathways behind the therapeutic effects of dirty GPCR modulators, and developing new agents with...