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Genetic Linkage

Two Newly-Approved Gene Therapies Cost Millions

The FDA recently approved two gene therapies with hefty price tags, the first for an inherited anemia and the second for a degenerative brain condition. The two new treatments, from bluebirdbio, double the number of gene therapies on the market.

 

Most biotechnologies evolve over three decades or so, but the idea of gene therapy has been around since the late 1950s, blooming soon after Watson and Crick solved the structure of DNA. When my book The Forever Fix: Gene Therapy and the Boy Who Saved It was published a decade ago, it would still be 5 years before the first approval. That treatment, the subject of my book, enabled the blind to see, sometimes in just days.

 

Why has the pace of gene therapy been so slow? Cost is one barrier. Other concerns are the degree to which a gene therapy actually helps, how long the effect lasts, and what proportion of patients respond.

 

A Short List

 

FDA's gene therapy roster is here, but a caveat is necessary.

 

 

To continue reading, go to DNA Science, where this post first appeared.

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Thoughts on a Return of Polio

When a case of polio showed up in Rockland county, just north of New York City, in July 2022, and then polioviruses with the same genetic sequence as from the paralyzed man were found in three samples of wastewater collected from near his home, public health officials were alarmed. The man, from an Orthodox Jewish community with low vaccination rates in general, had not been immunized against polio.

 

Definitions from the World Health Organization kicked in.

 

The viral RNA sequence from the patient was close to that of oral polio vaccine, which is "live" (weakened, aka attenuated). He was infected with vaccine-derived polioviruses (VDPV). Then finding the telltale RNA sequence in wastewater elevated the situation to circulating vaccine-derived polioviruses (cVDPV). The US now joins 30 other nations experiencing a return of this infectious disease that was once thought to be nearly gone.

 

To continue reading, go to DNA Science, where this post first appeared. 

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Mellow Yellow: A Biotech Way to Make Saffron

Biotechnology mass-produces valuable molecules from nature, from drugs to textiles to a jellyfish protein that lights up most anything a glowing green. Now add saffron to the list.

 

To cooks, saffron is a bright yellow spice derived from Crocus sativus flowers, aka "saffron crocus." The dried red threads at the blooms' centers are used to season and color foods. Popular for thousands of years, saffron comes today mostly from Iran. It's used to infuse meats, grains, salads, and even to color marshmallows molded into baby chick shapes for Easter.

 

Saffron has medicinal potential. The main pigment crocin may be useful as a neuroprotectant, an antidepressant, a sedative, and an antioxidant.

 

Inspiring a Song

To those of us of a certain age, the word "saffron" conjures up "mellow yellow," a 1966 song by Donovan:

 

I'm just mad about saffron
A-saffron's mad about me …
They call me mellow yellow …

 

To continue reading, go to DNA Science, where this post first appeared.

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Can Curcumin, Black Pepper, and Ginger Treat Retinitis Pigmentosa? Steve Fialkoff’s Excellent Experiment

Steve Fialkoff and I weren't friends at James Madison High School in Brooklyn, NY. We were in the class of 1972; earlier alums include Chuck Schumer, Bernie Sanders, and Carole King. The near-thousand of us self-sorted into three cliques, based on neighborhood. Steve was from Mill Basin, me Kings Highway.

 

While super-popular Steve was everywhere with his massive blond 'fro, capturing our experiences with his camera and leading the class in drama productions, I was on the fast track to nerddom. I spent my time in the chem lab with the groovy new teacher in charge, a 24-year-old who showed my bestie Wendy and me how to make bongs and water pipes. But Steve now says he was a closet nerd, a "frizzy-haired, freckle-faced, big-nosed, crooked-smile, toothy guy."

 

It wasn't surprising that Steve became a film editor and now a playwright. What was surprising was learning at age 25 that he had retinitis pigmentosa, after he tripped over a seat in a darkened theater and had a few other stumbles.

 

To continue reading, go to DNA Science, where this post first appeared.

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DNA Analysis Solves the Mystery of the Rabbit Invasion of Australia

COVID and monkeypox seem to have come out of nowhere and exploded across continents. But the phenomenon of natural selection acting on genetic variants – of viruses or organisms – that have an advantage in a certain place and time is ages old. The rabbits of Australia provide a powerful example of natural selection run amok, favoring a particularly robust mix of domestic and wild traits against an environmental backdrop of plenty of food and a paucity of predators.

 

The animals that have overrun the continent eat almost any plant, their appetites reverberating along food webs, costing an estimated $200 million a year. Over decades, interventions to control their numbers – from rabbit-proof fences to intentional infections with nasty viruses to shooting – have all failed. "In Australia, the rabbit has survived drought, fire, flood, diseases, predators, poisons and other stratagems devised by man and remains this country's most serious vertebrate pest," wrote Brian Coman in "Tooth and Nail: The Story of the Rabbit in Australia."

 

Now researchers from the University of Cambridge and CIBIO Institute in Portugal have wed genetics to history to illuminate the precise source of Australia's problem. Their report is in the Proceedings of the National Academy of Sciences.

 

To continue reading, go to DNA Science, where this post first appeared.

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