Researchers from Penn Medicine and Children’s Hospital of Philadelphia have fixed a lethal mutation in the prenatal mouse models of a rare pulmonary disease. The hope is that the approach of in utero editing described in Science Translational Medicine will work for other congenital lung diseases as well.
I asked one of the corresponding authors, Edward Morrison, scientific director of the Penn’s Institute for Regenerative Medicine, to explain what they have done and what to expect next. See Q&A below. Continue reading
It’s another CRISPR first: fixing a hereditary disease in utero. Scientists from Pennsylvania University and the Children’s Hospital of Philadelphia used a base editor to rescue tyrosinemia in fetal mice. “The results of this proof-of-concept work demonstrate the possibility of efficiently performing gene editing before birth, pointing to a potential new therapeutic approach for selected congenital disorders,” Avery C. Rossidis and colleagues write in 
“Uh Oh. CRISPR might not work on people”. A title like this on the 
The first patient 
Mosquito nets are not enough, vaccines are late to come, land reclamation in Africa is a challenge. But there is a new hope for defeating malaria, coming directly from the most advanced CRISPR frontier. The trick is a kind of genetic chain reaction fuelled by genetic elements called “gene drives”. Researchers are experimenting their power with the aim of crashing the number of mosquitoes responsible for Plasmodium transmission, by spreading genes that are bad for Anopheles gambiae. A gene behaving in Mendelian way has a 50% chance of being passed on from parent to offspring, but it can virtually reach 100% with a little help from a drive. Thus a gene designed to damage a harmful species can propagate within a few generations with a domino effect, until the population collapses. One of the founders of this futuristic strategy is an Italian molecular parasitologist: 
The aim is engaging: to treat an increasing number of diseases by correcting the underlying genetic defects. And researchers are breathing optimism at last. The San Raffaele Telethon Institute for Gene Therapy (
Elementary dear Watson, we should have expected that. The CRISPR wave is hitting diagnostics, with a new high sensitivity detection platform named after Arthur Conan Doyle’s popular detective. The acronym SHERLOCK stands for “Specific High Sensitivity Enzymatic Reporter UnLOCKing”. While the technique is used in thousands of labs to turn genes on and off, CRISPR embarks also on epidemiology and learns how to identify nucleic acids from viral and bacterial pathogens to diagnose infections. The paper published in 
CRISPeR FRENZY 