A gene therapy that makes use of an unlikely helper, the Aids virus, gave a working immune system to 48 babies and toddlers who were born without one, doctors reported on Tuesday.
Results show that all but two of the 50 children who were given the experimental therapy in a study now have healthy, germ-fighting abilities.
“We’re taking what otherwise would have been a fatal disease” and healing most of these children with a single treatment, said study leader Dr Donald Kohn of UCLA Mattel Children’s Hospital.
“They’re basically ‘free range’ – going to school, doing normal things” without the worry that any infection could become life-threatening, he said.
The other two children who were not helped by the gene therapy later had successful bone-marrow transplants. Doctors say it will take longer to know if any of the 50 are cured, but they seem to be doing well so far.
The children had severe combined immunodeficiency syndrome, or SCID, which is caused by an inherited genetic flaw that keeps the bone marrow from making healthy versions of the blood cells that form the immune system. Without treatment, it often kills in the first year or two of life.
It became known as "bubble boy disease" because of a case in the 1970s involving a boy in the US state of Texas who lived for 12 years in a protective plastic bubble to isolate him from germs. It is now called "bubble baby disease" because about 20 different gene defects, including some that affect girls as well as boys, can cause it.
A bone-marrow transplant from a genetically matched sibling can cure the disorder, but most children lack a suitable donor and the treatment is risky – the Texas boy died after one.
Patients now are treated with twice-weekly doses of antibiotics and germ-fighting antibodies, but it is not a permanent solution.
Doctors think gene therapy might be. They remove some of a patient’s blood cells, use a disabled Aids virus to insert a healthy version of the gene that the child needs, and return the cells through an IV.
Josselyn Kish, now 11 and living in Las Vegas, received the treatment at UCLA when she was three. As a baby, she suffered rashes, painful shingles and frequent diarrhoea, said her mother, Kim Carter. “Day care was calling me a couple of times a week to come get her because she was always getting fevers.”
After the gene therapy, "she was better right away", Ms Carter said. Now, "she rarely, rarely gets sick at all" and has been able to recover whenever she has. That hope extends to Josselyn's newest infection – she was recently found to have Covid-19 and so far has only very mild symptoms.
In all, 27 children were treated at the Los Angeles hospital, three at the US National Institutes of Health near Washington and 20 at Great Ormond Street Hospital in London. The fact the treatment seems safe across several hospitals performing it makes the study "very powerful", said Dr Stephen Gottschalk of St Jude Children's Research Hospital in Memphis, Tennessee.
He had no role in the new study but he and his colleagues have performed a similar gene therapy on 17 other children with SCID.
“People ask us, is it a cure? Who knows long term, but at least up to three years, these children are doing well,” Dr Gottschalk said. “The immune function seems stable over time, so I think it looks very, very encouraging.”
Results of the UCLA-led study were published on Tuesday by the New England Journal of Medicine and presented at an online American Society of Gene and Cell Therapy conference. Grants from US and British government health agencies and the tax-supported California Institute for Regenerative Medicine paid for the work. Dr Kohn is an inventor of the treatment and an adviser to the company now developing it, London-based Orchard Therapeutics.
Benefits of first-time home buyers' scheme
- Priority access to new homes from participating developers
- Discounts on sales price of off-plan units
- Flexible payment plans from developers
- Mortgages with better interest rates, faster approval times and reduced fees
- DLD registration fee can be paid through banks or credit cards at zero interest rates
Squid Game season two
Director: Hwang Dong-hyuk
Stars: Lee Jung-jae, Wi Ha-joon and Lee Byung-hun
Rating: 4.5/5
More from Neighbourhood Watch
COMPANY%20PROFILE
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FIXTURES
Nov 04-05: v Western Australia XI, Perth
Nov 08-11: v Cricket Australia XI, Adelaide
Nov 15-18 v Cricket Australia XI, Townsville (d/n)
Nov 23-27: 1ST TEST v AUSTRALIA, Brisbane
Dec 02-06: 2ND TEST v AUSTRALIA, Adelaide (d/n)
Dec 09-10: v Cricket Australia XI, Perth
Dec 14-18: 3RD TEST v AUSTRALIA, Perth
Dec 26-30 4TH TEST v AUSTRALIA, Melbourne
Jan 04-08: 5TH TEST v AUSTRALIA, Sydney
Note: d/n = day/night
The National's picks
4.35pm: Tilal Al Khalediah
5.10pm: Continous
5.45pm: Raging Torrent
6.20pm: West Acre
7pm: Flood Zone
7.40pm: Straight No Chaser
8.15pm: Romantic Warrior
8.50pm: Calandogan
9.30pm: Forever Young
HOW TO WATCH
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Company profile
Name: Steppi
Founders: Joe Franklin and Milos Savic
Launched: February 2020
Size: 10,000 users by the end of July and a goal of 200,000 users by the end of the year
Employees: Five
Based: Jumeirah Lakes Towers, Dubai
Financing stage: Two seed rounds – the first sourced from angel investors and the founders' personal savings
Second round raised Dh720,000 from silent investors in June this year
ZAYED SUSTAINABILITY PRIZE
MATCH RESULT
Liverpool 4 Brighton and Hove Albion 0
Liverpool: Salah (26'), Lovren (40'), Solanke (53'), Robertson (85')
The specs
Engine: 2.0-litre 4-cylinder turbo
Power: 240hp at 5,500rpm
Torque: 390Nm at 3,000rpm
Transmission: eight-speed auto
Price: from Dh122,745
On sale: now
Mohammed bin Zayed Majlis
if you go
The flights
Fly direct to Kutaisi with Flydubai from Dh925 return, including taxes. The flight takes 3.5 hours. From there, Svaneti is a four-hour drive. The driving time from Tbilisi is eight hours.
The trip
The cost of the Svaneti trip is US$2,000 (Dh7,345) for 10 days, including food, guiding, accommodation and transfers from and to Tbilisi or Kutaisi. This summer the TCT is also offering a 5-day hike in Armenia for $1,200 (Dh4,407) per person. For further information, visit www.transcaucasiantrail.org/en/hike/
The biog
Favourite book: Homegoing by Yaa Gyasi
Favourite holiday destination: Spain
Favourite film: Bohemian Rhapsody
Favourite place to visit in the UAE: The beach or Satwa
Children: Stepdaughter Tyler 27, daughter Quito 22 and son Dali 19
Jetour T1 specs
Engine: 2-litre turbocharged
Power: 254hp
Torque: 390Nm
Price: From Dh126,000
Available: Now
THE BIO
Born: Mukalla, Yemen, 1979
Education: UAE University, Al Ain
Family: Married with two daughters: Asayel, 7, and Sara, 6
Favourite piece of music: Horse Dance by Naseer Shamma
Favourite book: Science and geology
Favourite place to travel to: Washington DC
Best advice you’ve ever been given: If you have a dream, you have to believe it, then you will see it.
Francesco Totti's bio
Born September 27, 1976
Position Attacking midifelder
Clubs played for (1) - Roma
Total seasons 24
First season 1992/93
Last season 2016/17
Appearances 786
Goals 307
Titles (5) - Serie A 1; Italian Cup 2; Italian Supercup 2
Credit Score explained
What is a credit score?
In the UAE your credit score is a number generated by the Al Etihad Credit Bureau (AECB), which represents your credit worthiness – in other words, your risk of defaulting on any debt repayments. In this country, the number is between 300 and 900. A low score indicates a higher risk of default, while a high score indicates you are a lower risk.
Why is it important?
Financial institutions will use it to decide whether or not you are a credit risk. Those with better scores may also receive preferential interest rates or terms on products such as loans, credit cards and mortgages.
How is it calculated?
The AECB collects information on your payment behaviour from banks as well as utilitiy and telecoms providers.
How can I improve my score?
By paying your bills on time and not missing any repayments, particularly your loan, credit card and mortgage payments. It is also wise to limit the number of credit card and loan applications you make and to reduce your outstanding balances.
How do I know if my score is low or high?
By checking it. Visit one of AECB’s Customer Happiness Centres with an original and valid Emirates ID, passport copy and valid email address. Liv. customers can also access the score directly from the banking app.
How much does it cost?
A credit report costs Dh100 while a report with the score included costs Dh150. Those only wanting the credit score pay Dh60. VAT is payable on top.
SPEC%20SHEET%3A%20APPLE%20IPAD%20(2022)
%3Cp%3E%3Cstrong%3EDisplay%3A%3C%2Fstrong%3E%2010.9-inch%20Liquid%20Retina%20IPS%20LCD%2C%202%2C360%20x%201%2C640%2C%20264ppi%2C%20wide%20colour%2C%20True%20Tone%2C%20Apple%20Pencil%201%20support%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EChip%3A%3C%2Fstrong%3E%20Apple%20A14%20Bionic%2C%206-core%20CPU%2C%204-core%20GPU%2C%2016-core%20Neural%20Engine%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EStorage%3A%3C%2Fstrong%3E%2064GB%2F256GB%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EPlatform%3A%3C%2Fstrong%3E%20iPadOS%2016%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EMain%20camera%3A%3C%2Fstrong%3E%2012-megapixel%20wide%2C%20f%2F1.8%2C%205x%20digital%2C%20Smart%20HDR%203%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EVideo%3A%3C%2Fstrong%3E%204K%20%40%2024%2F25%2F30%2F60fps%2C%20full%20HD%20%40%2025%2F30%2F60fps%2C%20slo-mo%20%40%20120%2F240fps%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EFront%20camera%3A%3C%2Fstrong%3E%2012MP%20ultra-wide%2C%20f%2F2.4%2C%202x%2C%20Smart%20HDR%203%2C%20Centre%20Stage%3B%20full%20HD%20%40%2025%2F30%2F60fps%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EAudio%3A%3C%2Fstrong%3E%20Stereo%20speakers%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EBiometrics%3A%3C%2Fstrong%3E%20Touch%20ID%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EI%2FO%3A%3C%2Fstrong%3E%20USB-C%2C%20smart%20connector%20(for%20folio%2Fkeyboard)%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EBattery%3A%3C%2Fstrong%3E%20Up%20to%2010%20hours%20on%20Wi-Fi%3B%20up%20to%209%20hours%20on%20cellular%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EFinish%3A%3C%2Fstrong%3E%20Blue%2C%20pink%2C%20silver%2C%20yellow%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EIn%20the%20box%3A%3C%2Fstrong%3E%20iPad%2C%20USB-C-to-USB-C%20cable%2C%2020W%20power%20adapter%3C%2Fp%3E%0A%3Cp%3E%3Cstrong%3EPrice%3A%3C%2Fstrong%3E%20Wi-Fi%20%E2%80%94%20Dh1%2C849%20(64GB)%20%2F%20Dh2%2C449%20(256GB)%3B%20cellular%20%E2%80%94%20Dh2%2C449%20(64GB)%20%2F%20Dh3%2C049%20(256GB)%3C%2Fp%3E%0A
India squad for fourth and fifth Tests
Kohli (c), Dhawan, Rahul, Shaw, Pujara, Rahane (vc), Karun, Karthik (wk), Pant (wk), Ashwin, Jadeja, Pandya, Ishant, Shami, Umesh, Bumrah, Thakur, Vihari
Know your Camel lingo
The bairaq is a competition for the best herd of 50 camels, named for the banner its winner takes home
Namoos - a word of congratulations reserved for falconry competitions, camel races and camel pageants. It best translates as 'the pride of victory' - and for competitors, it is priceless
Asayel camels - sleek, short-haired hound-like racers
Majahim - chocolate-brown camels that can grow to weigh two tonnes. They were only valued for milk until camel pageantry took off in the 1990s
Millions Street - the thoroughfare where camels are led and where white 4x4s throng throughout the festival
Key findings of Jenkins report
- Founder of the Muslim Brotherhood, Hassan al Banna, "accepted the political utility of violence"
- Views of key Muslim Brotherhood ideologue, Sayyid Qutb, have “consistently been understood” as permitting “the use of extreme violence in the pursuit of the perfect Islamic society” and “never been institutionally disowned” by the movement.
- Muslim Brotherhood at all levels has repeatedly defended Hamas attacks against Israel, including the use of suicide bombers and the killing of civilians.
- Laying out the report in the House of Commons, David Cameron told MPs: "The main findings of the review support the conclusion that membership of, association with, or influence by the Muslim Brotherhood should be considered as a possible indicator of extremism."
