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Genetic Linkage

Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). Available in Italy for three years, Lenmeldy (atidarsagene autotemcel), from Orchard Therapeutics, is groundbreaking, but comes at quite a cost – the $4.25 million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it.

 

An Ultrarare Neurological Condition

 

MLD affects the white matter in the brain, causing progressive loss of mobility and sensation, as well as intellectual decline and, ultimately, unresponsiveness.

 

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

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Cultivated Meat? Let Them Eat Snake

Biotechnology has solved many problems, from recombinant DNA and monoclonal antibody-derived drugs, to gene therapy and stem cell transplants, to RNA-based vaccines and genetically modified plants that resist diseases and pesticides.

 

In contrast, so-called cultivated meat has been, so far, a failure.

 

Joe Fassler's in-depth Opinion piece in the February 9 New York Times, The Revolution That Died on Its Way to Dinner, digests the unrealistic expectations, shortcuts, and glitches that have stymied what he envisions as "a high-tech factory housing steel tanks as tall as apartment buildings and conveyor belts rolling out fully formed steaks, millions of pounds a day — enough, astonishingly, to feed an entire nation."

 

Making Meat

 

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

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How the Human Lost Its Tail

In 1902's Just So Stories for Little Children, British author Rudyard Kipling famously explained curiosities of the animal kingdom: How the Leopard Got His Spots, How the Camel got his Hump, How the Rhinoceros got his Skin, to name a few.

 

Reading Just So Stories was one of my earliest memories of thinking like a scientist. I see them in articles on animals' oddities, such as How the Tabby Got its Stripes, in which I explored a molecular explanation for fur color patterns set in the fetus, from a report in Nature Communications.

 

Now new research published in Nature brings the just-so approach to the loss of tails among apes – including us.

 

Apes R Us

 

Whether or not taillessness was a liability as we evolved depends upon perspective and imagination. Would absence of a fifth appendage have made walking erect – bipedalism – easier? All mammals other than apes have tails, if only as embryos, which is the case for humans. Our tailbones are the remnants of tails.

 

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

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“Ordinary Soil” Revisits the Weedkiller and AgBiotech Story, While Feeding the Scientist-As-Nerd Stereotype

I love the spectacular symbiosis of my vegetable garden as harvest time approaches.

 

Beanstalks spiral up cornstalks, their tendrils teasing nearby tomato stems. Below, broad leaves protect ballooning squashes from the slugs that appear, seemingly from nowhere, after a rain, while providing water for passing furry creatures.

 

The synergism of a garden is an ancient and somewhat obvious idea. Many indigenous peoples honored the "three sisters" of corn, beans, and squash. My kids – three sisters – learned about the practice in grade school, and all recall the first meal that we grew: corn, beans, and squash.

 

Those memories returned as I read Alex Woodard's excellent novel Ordinary Soil.

 

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

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