US FDA approves Mirum Pharma's genetic disorder drug
The U.S. Food and Drug Administration approved Mirum Pharmaceuticals' genetic disorder drug on Friday, making it the first ...
In vitro reconstitution of meiotic DNA double-strand breaks: A long-standing challenge solved
In a study published in Nature, researchers report the successful in vitro reconstitution of meiotic DNA double-strand breaks ...
How do cells respond to changes? New study finds it's not all in the genes
Cells are constantly on the move, whether in a developing embryo or metastatic cancer. But how do cells adapt to the new ...
The Economist12h
Gene editing has put biological research on a new trajectory
When the monkeys were single-celled embryos, scientists had used CRISPR editing tools to silence, or “knock out”, a gene that ...
Characterizing the spectrum of mitochondrial DNA deletion disease syndromes
Researchers from the Mitochondrial Medicine Program at Children's Hospital of Philadelphia (CHOP) have better characterized a spectrum of what were classically considered discrete mitochondrial DNA ...
Researchers identify novel gene behind neurodevelopmental syndrome for enhanced clinical treatment
A clinical research team from the LKS Faculty of Medicine, the University of Hong Kong (HKUMed), and international genetic researchers led a global research study using multi-omics analysis and ...
The Economist12h
CRISPR cures face cost and safety concerns
Doctors harvest stem cells from the patient’s bone marrow and then send them off to a lab, where CRISPR is used to turn up the production of another, functional version of haemoglobin. This version is ...
Live Science on MSN12h
Deadly motor-neuron disease treated in the womb in world 1st
An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...