Game-changing genetic editing just let one teenager 'dodge' sickle cell disease.

Chalk up another big potential win for science – it just helped one kid avoid some of the devastating effects of sickle cell disease.

Sickle cell disease is a chronic, genetic blood disorder that affects about 100,000 Americans. It can affect anyone, although it disproportionately occurs in African-Americans. It’s manageable with medicine and proper care, but can still be dangerous and extremely painful. It's a lifelong condition.

Or, maybe not. Because that last part — about it being a lifelong condition – might be changing.


A French teenager may have just dodged most of the effects of the disorder thanks to gene editing.

A worker preparing cells for gene editing research. Ben Birchall/PA via AP.

That's according to a March 2, 2017, report in The New England Journal of Medicine.

Sickle cell is caused by a single genetic typo in the gene that produces hemoglobin — an important blood protein. That typo can cause normally disc-shaped red blood cells to warp into sickle-shaped crescents.

On the right are normal-shaped red blood cells. On the left is one deformed into the classic sickle shape. Image from Sickle Cell Foundation of Georgia.

These can get trapped in small blood vessels, causing painful, sometimes dangerous blood clots.

The researchers behind the study used gene editing to install a typo-free copy of the gene into the DNA of some of the boy's cells.

A modified virus shepherded a copy of the normal hemoglobin gene into the boy's bone marrow stem cells (which produce red blood cells).

Now, more than two years later, about half of the boy’s red blood cells are carrying that normal protein.

"It’s not a cure, but it doesn’t matter," explained Philippe Leboulch, who helped invent the therapy, because the results were enough for the patient to avoid most of the effects of sickle cell.

Other gene therapy studies for sickle cell are also underway in Los Angeles and California.

The treatment is still preliminary work, but if it holds out in the long run, it could be a huge win for a lot of people.

In Indianapolis, a newborn's blood is taken to test for inherited diseases such as sickle cell. Photo from AP Photo/Michael Conroy.

"It could be a game changer," Oxford geneticist Deborah Gill told New Scientist. "The fact the team has a patient with real clinical benefit, and biological markers to prove it, is a very big deal."

In the past, researchers have had success fighting sickle cell with stem cell transplants, but finding a compatible donor can be difficult for many patients. Gene therapy, however, only needs one person — the patient.

Of course, there's still a ways to go before this could be widely available. But if it works out, this technique could help over 100,000 Americans live without the chronic pain of sickle cell. Which is the very definition of "a very big deal."

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A lot of people here are like family to me," Michelle says about Bread for the City — a community nonprofit located in Washington DC that provides local residents with food, clothing, health care, social advocacy, and legal services. And since the pandemic began, the need to support organizations like Bread for the City is greater than ever, which is why Amazon is Delivering Smiles to local charities across the country this holiday season.

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Amazon is giving back by fulfilling hundreds of AmazonSmile Charity Lists, and donating essential pantry and food items to help organizations like Bread for the City provide to those disproportionately impacted this year.

Visit AmazonSmile Charity Lists to donate directly to a local charity in your community, or simply shop smile.amazon.com and Amazon will donate a portion of the purchase price of eligible products to your charity of choice.

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Courtesy of Macy's

Brantley and his snowman

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"Would you like to build a snowman?" If you asked five-year-old Brantley from Texas this question, the answer would be a resounding "Yes!" While it may sound like a simple dream, since Texas doesn't usually see much snow, it seemed like a lofty one for him, even more so because Brantley has a congenital heart disease.

On Dec. 11, 2019, however, the real Macy's Santa and his two elves teamed up with Make-A-Wish to surprise Brantley and his family on his way to Colorado where there was plenty of snow for him to build his very own snowman, fulfilling his wish as part of the Macy's Believe campaign. After a joy-filled plane ride where every passenger got gift bags from Macy's, the family arrived in Breckenridge, Colorado where Santa and his elves helped Brantley build a snowman.

Brantley, Brantley's mom, and Santa marveling at their snowmanAll photos courtesy of Macy's

Brantley, who according to his mom had never actually seen snow, was blown away by the experience.

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This is just one example of the more than 330,000 wishes the nonprofit Make-A-Wish have fulfilled to bring joy to children fighting critical illnesses since its founding 40 years ago. Even though many of the children that Make-A-Wish grants wishes for manage or overcome their illnesses, they often face months, if not years of doctor's visits, hospital stays and uncomfortable treatments. The nonprofit helps these children and their families replace fear with confidence, sadness with joy and anxiety with hope.

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Brantley playing with snowballs

Now more than ever, kids with critical illnesses need hope. Since they're particularly vulnerable to disease, they and their families have had to isolate even more during the pandemic and avoid the people they love most and many of the activities that recharge them. That's why Make-A-Wish is doing everything it can to fulfill wishes in spite of the unprecedented obstacles.

That's where you come in. Macy's has raised over $132 million for Make-A-Wish, and helped grant more than 15,500 wishes since their partnership began in 2003, but they couldn't have done that without the support of everyday people. The crux of that support comes from Macy's Believe Campaign — the longstanding holiday fundraising effort where for every letter to Santa that's written online at Macys.com or dropped off safely at the red Believe mailbox at their stores, Macy's will donate $1 to Make-A-Wish, up to $1 million. New this year, National Believe Day will be expanded to National Believe Week and will provide customers the opportunity to double their donations ($2 per letter, up to an additional $1 million) for a full week from Sunday, Nov. 29 through Saturday, Dec. 5.

There are more ways to support Make-A-Wish besides letter-writing too. If you purchase a $4 Believe bracelet, $2 of each bracelet will be donated to Make-A-Wish through Dec. 31. And for families who are all about the holiday PJs, on Giving Tuesday (Dec. 1), 20 percent of the purchase price of select family pajamas will benefit Make-A-Wish.

Elizabeth living out her wish of being a fashion designer

Additionally, this year's campaign features 6-year-old Elizabeth, a Make-A-Wish child diagnosed with leukemia, whose wish to design a dress recently came true. Thanks to the style experts at Macy's Fashion Office and I.N.C. International Concepts, only at Macy's, Elizabeth had the opportunity to design a colorful floral maxi dress. Elizabeth's exclusive design is now available online at Macys.com and in select Macy's stores. In the spirit of giving back this holiday season, 20 percent of the purchase price of Elizabeth's dress (through Dec. 31) will benefit Make-A-Wish.You can also donate directly to Make-A-Wish via Macy's website.

This holiday season may be a tough one this year, but you can bring joy to children fighting critical illnesses by delivering hope for their wishes to come true.

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