Babies born with infantile-onset Pompe disease typically have enlarged hearts and weak muscles. But 1-year-old Ayla has a normal heart and walks.

A toddler is thriving after doctors in the U.S. and Canada used a novel technique to treat her before she was born for a rare genetic disease that caused the deaths of two of her sisters.

Ayla Bashir, a 16-month-old from Ottawa, Ontario, is the first child treated as a fetus for Pompe disease, an inherited and often fatal disorder in which the body fails to make some or all of a crucial protein.

The human genome has just over 20,000 genes coding for proteins. Yet, it produces at least ten times that many different non-coding RNA molecules, which can often take on more than one shape. At least some of this RNA structurome is functional in physiology or pathophysiology.

In an invited review for Nature Reviews Genetics, Danny Incarnato, a molecular geneticist from the University of Groningen (The Netherlands), and his colleague Robert C. Spitale from the University of Irvine in California (USA) describe ways to develop the, as yet, largely untapped potential of RNA structures.

RNA is perhaps best known as the intermediate between genome and protein synthesis: messenger RNA molecules copy the genetic code of a gene in the cell’s nucleus and transport it to the cytoplasm, where ribosomes translate the code into a protein. However, RNA is also a key regulator of almost every cellular process and the structures that are adopted by RNA molecules are thought to often be key to their functions.

Based on marketing activation events the company ran over the summer in Seattle, Austin, and Palo Alto, the outlook for their first product looks pretty rosy. They gave away bags of salad (which were clearly labeled as being gene-edited) consisting of red-and green-leaf mustard greens, and asked people to complete a short survey about it. Adams estimated that more than 6,000 people tried the salads, and over 90 percent responded that they were “very motivated” or “somewhat motivated” to buy the product.

A New Green Revolution?

Helping people make healthier dietary choices is just one benefit that CRISPR could bring to produce. Its possibilities are wide-ranging, as evidenced by PairWise’s work to create fruit trees that can grow in different climates and yield food that’s easier to harvest. It’s not unlike Norman Borlaug’s work back in the 1940s to create a high-yield wheat seed that was resistant to stem rust—a project that ended up saving millions of people from hunger and famine.

Ribonucleic acid (RNA) is a polymeric molecule similar to DNA that is essential in various biological roles in coding, decoding, regulation and expression of genes. Both are nucleic acids, but unlike DNA, RNA is single-stranded. An RNA strand has a backbone made of alternating sugar (ribose) and phosphate groups. Attached to each sugar is one of four bases—adenine (A), uracil (U), cytosine ©, or guanine (G). Different types of RNA exist in the cell: messenger RNA (mRNA), ribosomal RNA (rRNA), and transfer RNA (tRNA).” RNA is an important information transmitter in our cells and acts as a blueprint for protein production. When freshly formed RNA is processed, introns are removed to produce mature mRNA coding for protein. This cutting is known as “splicing,” and it is controlled by a complex known as the “spliceosome.”

“We found a gene in worms, called PUF60, that is involved in RNA splicing and regulates life span,” says Max Planck scientist Dr. Wenming Huang who made the discovery.

This gene’s mutations resulted in inaccurate splicing and the retention of introns within certain RNAs. As a result, less of the corresponding proteins were produced from this RNA. Surprisingly, worms with the PUF60 gene mutation survived significantly longer than normal worms.