PROMISING GENE THERAPY DELIVERS TREATMENT DIRECTLY TO BRAIN
Techlife News|January 28, 2023
When Rylae-Ann Poulin was a year old, she didn’t crawl or babble like other kids her age. A rare genetic disorder kept her from even lifting her head. Her parents took turns holding her upright at night just so she could breathe comfortably and sleep.
PROMISING GENE THERAPY DELIVERS TREATMENT DIRECTLY TO BRAIN

Then, months later, doctors delivered gene therapy directly to her brain.

Now the 4-year-old is walking, running, swimming, reading and riding horses — “just doing so many amazing things that doctors once said were impossible,” said her mother, Judy Wei.

Rylae-Ann, who lives with her family in Bangkok, was among the first to benefit from a new way of delivering gene therapy — attacking diseases inside the brain — that experts believe holds great promise for treating a host of brain disorders.

Her treatment recently became the first brain-delivered gene therapy after its approval in Europe and the United Kingdom for AADC deficiency, a disorder that interferes with the way cells in the nervous system communicate. New Jersey drugmaker PTC Therapeutics plans to seek U.S. approval this year.

Meanwhile, about 30 U.S. studies testing gene therapy to the brain for various disorders are ongoing, according to the National Institutes of Health. One, led by Dr. Krystof Bankiewicz at Ohio State University, also targets AADC deficiency. Others test treatments for disorders such as Alzheimer’s, Parkinson’s and Huntington’s.

Challenges remain, especially with diseases caused by more than a single gene. But scientists say the evidence supporting this approach is mounting — opening a new frontier in the fight against disorders afflicting our most complex and mysterious organ.

“There’s a lot of exciting times ahead of us,” said Bankiewicz, a neurosurgeon. “We’re seeing some breakthroughs.”

Bu hikaye Techlife News dergisinin January 28, 2023 sayısından alınmıştır.

Start your 7-day Magzter GOLD free trial to access thousands of curated premium stories, and 9,000+ magazines and newspapers.

Bu hikaye Techlife News dergisinin January 28, 2023 sayısından alınmıştır.

Start your 7-day Magzter GOLD free trial to access thousands of curated premium stories, and 9,000+ magazines and newspapers.

TECHLIFE NEWS DERGISINDEN DAHA FAZLA HIKAYETümünü görüntüle
SOUTH KOREA FINES META $15 MILLION FOR ILLEGALLY COLLECTING INFORMATION ON FACEBOOK USERS
Techlife News

SOUTH KOREA FINES META $15 MILLION FOR ILLEGALLY COLLECTING INFORMATION ON FACEBOOK USERS

South Korea’s privacy watchdog this week fined social media company Meta 21.6 billion won ($15 million) for illegally collecting sensitive personal information from Facebook users, including data about their political views and sexual orientation, and sharing it with thousands of advertisers.

time-read
2 dak  |
November 09, 2024
CHINESE ONLINE RETAILER TEMU FACES EUROPEAN UNION INVESTIGATION INTO ROGUE TRADERS AND ILLEGAL GOODS
Techlife News

CHINESE ONLINE RETAILER TEMU FACES EUROPEAN UNION INVESTIGATION INTO ROGUE TRADERS AND ILLEGAL GOODS

Chinese online retailer Temu is facing a European Union investigation over suspicions it’s failing to prevent the sale of illegal products, the 27-nation bloc’s executive arm said.

time-read
2 dak  |
November 09, 2024
CORNING FACES EU ANTITRUST SCRUTINY OVER GORILLA GLASS DEALS WITH PHONE MAKERS
Techlife News

CORNING FACES EU ANTITRUST SCRUTINY OVER GORILLA GLASS DEALS WITH PHONE MAKERS

Corning is facing European Union antitrust scrutiny after the bloc’s watchdog said this week it’s investigating the speciality glass maker’s exclusive deals for its Gorilla Glass used in cellphones.

time-read
1 min  |
November 09, 2024
NEW CEO AT CVS BEGINS TO BUILD HIS TEAM WITH A GOAL OF REVIVING THE STRUGGLING HEALTH CARE GIANT
Techlife News

NEW CEO AT CVS BEGINS TO BUILD HIS TEAM WITH A GOAL OF REVIVING THE STRUGGLING HEALTH CARE GIANT

CVS Health fell short on third-quarter profit, but it posted strong sales and the health care giant shook up leadership under new CEO David Joyner after a rough year that has sent shares plunging.

time-read
2 dak  |
November 09, 2024
NINTENDO REPORTS LOWER PROFITS AS DEMAND DROPS FOR ITS AGING SWITCH CONSOLE
Techlife News

NINTENDO REPORTS LOWER PROFITS AS DEMAND DROPS FOR ITS AGING SWITCH CONSOLE

Nintendo, the Japanese video game maker behind the Super Mario franchise, said Tuesday that its profit fell 60% in the first half of the fiscal year, as demand waned for its Switch console, now in its eighth year since going on sale.

time-read
1 min  |
November 09, 2024
AS THE DATA CENTER INDUSTRY BOOMS, AN ENGLISH VILLAGE BECOMES A BATTLEGROUND
Techlife News

AS THE DATA CENTER INDUSTRY BOOMS, AN ENGLISH VILLAGE BECOMES A BATTLEGROUND

Originally built to store crops from peasant farmers, the Tithe Barn on the edge of the English village of Abbots Langley was converted into homes that preserve its centuries of history.

time-read
4 dak  |
November 09, 2024
THE BEST SMALL SUVS FOR CITY DRIVING
Techlife News

THE BEST SMALL SUVS FOR CITY DRIVING

SUVs are popular in part due to their elevated ride height, commanding view of the road, and roomy interiors.

time-read
3 dak  |
November 09, 2024
GOOGLE MAPS ADDS AI FEATURES TO HELP USERS EXPLORE AND NAVIGATE THE WORLD AROUND THEM
Techlife News

GOOGLE MAPS ADDS AI FEATURES TO HELP USERS EXPLORE AND NAVIGATE THE WORLD AROUND THEM

Google Maps is heading down a new road steered by artificial intelligence.

time-read
2 dak  |
November 09, 2024
TESLA SHARES SOAR 14% AS TRUMP WIN SETS STAGE FOR ELON MUSK'S ELECTRIC VEHICLE COMPANY
Techlife News

TESLA SHARES SOAR 14% AS TRUMP WIN SETS STAGE FOR ELON MUSK'S ELECTRIC VEHICLE COMPANY

Shares of Tesla soared following an election that will send Donald Trump back to the White House, an outcome that has been strongly backed by CEO Elon Musk in the closing months of the race.

time-read
1 min  |
November 09, 2024
APPLE BREAKS OUT OF RECENT SALES SLUMP AS IT GEARS UP TO MAKE ITS LEAP INTO THE AI CRAZE
Techlife News

APPLE BREAKS OUT OF RECENT SALES SLUMP AS IT GEARS UP TO MAKE ITS LEAP INTO THE AI CRAZE

Apple snapped out of a recent iPhone sales slump during its summer quarter, an early sign that its recent efforts to revive demand for its marquee product with an infusion of artificial intelligence are paying off.

time-read
2 dak  |
November 09, 2024