Gene therapy targets sickle-cell disease – Nature

Image credit: Steve Babuljak/UCSF

Gene therapy might offer a cure for sickle-cell disease, and clinical trials are already under way. The approach is promising because just a single gene needs correcting: the one for the β-globin subunit of haemoglobin, the body’s oxygen ferry. But Elliott Vichinsky is concerned that the same problems that make current sickle-cell care ineffective will also plague this gene-therapy treatment. He estimates that at least 30% of his adult patients with sickle-cell disease die from preventable causes. As his patients attest, sickle-cell care is often inadequate for reasons that have little to do with scientific advancement and lots to do with economics and racism.

 

How cerebral organoids are guiding brain-cancer research and therapies – Nature

Image credit: M. Lancaster/MRC-LMB

People with glioblastoma multiforme, one of the most common forms of brain cancer, have a median survival of less than 15 months after diagnosis. If researchers could grow numerous small brain-like structures that contained a replica of the person’s tumour and then bathe them in various treatments, in the space of a few weeks, they might learn exactly which ones would have the best chance of fighting brain cancer in that individual. Howard Fine, a neuro-oncologist at Weill Cornell Medicine in New York City, is developing such models, known as cerebral organoids. Organoids are particularly valuable for studying brain cancer because neither human brain tumours transplanted into mice nor human tumour stem cells grown in a culture dish behave in the same way as their counterparts in the body.

What will it take to 3-D print organs? – NeoLife

Sebastian Kaulitzki/Shutterstock; Scientific Reports

Every day in the U.S., about 22 people die waiting for an organ transplant. If scientists could 3-D print organs like kidneys, livers and hearts, all those lives could be saved. For years, people have been touting personalized organ printing as the future.

But despite decades of promising work in bioengineered bladders and other kinds of human tissue, we’re not close to having more complicated organs made from scratch. Harvard professor Jennifer Lewis, a leader in advanced 3-D printing of biological tissue, has only recently developed the ability to print part of a nephron, an individual unit of a kidney.

I asked Lewis what it will take to someday print a full kidney or a similarly complex organ.

Read the full story in NeoLife.

Take two placebos and call me in the morning – NeoLife

Illustration by Gabriel Alcala

When a new drug is being tested in a controlled clinical trial, half the patients get the real drug and half get a placebo, something harmless like a sugar pill or a saline injection. But patients on the placebo often improve anyway, and that’s because they expect that they’re getting the real drug, right? Well, no. Harvard professor Ted Kaptchuk’s research has exploded that explanation. Read the full story in NeoLife.

Gene-Editing Startup Raises $120 Million to Apply CRISPR to Medicine

Editas Medicine, a company at the forefront of developing the gene-editing technology known as CRISPR, has raised $120 million to create new treatments for conditions including cancer, retinal disease, and sickle-cell anemia.

Monday’s announcement reflects a surge of interest in CRISPR, a technology that is only a few years old. It also serves to clarify the goals and strategy of Editas, which was founded by some of the most prominent inventors of the gene-editing system, including Feng Zhang, a researcher at the Broad Institute of Harvard and MIT.

Read the full article in MIT Technology Review.