The Food and Drug Administration approved the first treatment for the most common cause of dwarfism Friday, a drug that has proved to increase children’s height but has been polarizing among adults with short stature.

The treatment, developed by BioMarin Pharmaceutical, is a once-daily injection for children with achondroplasia, a rare genetic disorder that results in dwarfism and can lead to serious medical complications. In a pivotal clinical trial, patients who got the drug, called Voxzogo, grew 1.6 centimeters more over the course of a year than those who received placebo. That means patients who take Voxzogo throughout childhood are likely to reach heights similar to their peers who don’t have achondroplasia, according to BioMarin.

“It’s the difference between being able to drive a car or not, reaching stuff in closets, being able to take care of your hygiene,” said Jean-Jacques Bienaimé, BioMarin’s CEO. “It would make a huge difference for those patients. There’s no question about it.”

The development of a genetic code expansion-based system enables fast protein expression in response to a noncanonical amino acid. The system was implanted into diabetic mice to rescue hyperglycemia with oral delivery of the amino acid.

In an unprecedented atlas, researchers begin to map how genes are turned on or off in different cells, a step toward better understanding the connections between genetics and disease.

Researchers at University of California San Diego have produced a single-cell chromatin atlas for the human genome. Chromatin is a complex of DNA

DNA, or deoxyribonucleic acid, is a molecule composed of two long strands of nucleotides that coil around each other to form a double helix. It is the hereditary material in humans and almost all other organisms that carries genetic instructions for development, functioning, growth, and reproduction. Nearly every cell in a person’s body has the same DNA. Most DNA is located in the cell nucleus (where it is called nuclear DNA), but a small amount of DNA can also be found in the mitochondria (where it is called mitochondrial DNA or mtDNA).

A model of facilitated diffusion and the theory of Anderson localization help explain how the Cas9 protein explores DNA in search of its targets.

The problem? Our bodies aren’t big fans of foreign substances—particularly ones that trigger an undesirable immune response. What’s more, these delivery systems aren’t great with biological zip codes, often swarming the entire body instead of focusing on the treatment area. These “delivery problems” are half the battle for effective genetic medicine with few side effects.

“The biomedical community has been developing powerful molecular therapeutics, but delivering them to cells in a precise and efficient way is challenging,” said Zhang at the Broad Institute, the McGovern Institute, and MIT.

Enter SEND. The new delivery platform, described in Science, dazzles with its sheer ingenuity. Rather than relying on foreign carriers, SEND (selective e ndogenous e n capsidation for cellular delivery) commandeers human proteins to make delivery vehicles that shuttle in new genetic elements. In a series of tests, the team embedded RNA cargo and CRISPR components inside cultured cells in a dish. The cells, acting as packing factories, used human proteins to encapsulate the genetic material, forming tiny balloon-like vessels that can be collected as a treatment.