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The Latest Breaking News from Gene Therapy

Gene Therapy is Upending How Doctors Treat Incurable Conditions

Gene therapy has helped doctors make major strides in treating inherited blindness, and it's now being explored for other conditions.

Inverse

Gene therapy helps combat some forms of blindness – and ongoing clinical trials are looking to extend these treatments to other diseases

An estimated 295 million people suffer from visual impairment globally. Around 43 million of those people are living with blindness. While not every form of blindness can be cured, recent scientific breakthroughs have uncovered new ways to treat some forms of inherited blindness through gene therapy.

The Conversation

'People say we are playing God changing our daughter's genetics - but it saved her'

Nicola Kelleher's partner Bradley Benjamin has a hereditary kidney disease which has seen him suffer on dialysis for years - and they didn't want the same outcome for their child

Daily Mirror

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Gene therapy for muscular dystrophy stirs hopes and controversy

The FDA is considering greenlighting the experimental treatment under its accelerated approvals program. Some critics point out the therapy isn't yet proven to work and may be costly.

WEKU

Genetic therapy can lower tau protein which causes Alzheimer's disease: Study

A world-first trial at UCL and UCLH has found a new gene therapy for Alzheimer's disease that is able to safely lower levels of the harmful tau protein known to cause the disease. Read further on Dynamite News:

Dynamite News

Gene therapy: Everything you need to know about the DNA-tweaking treatments

Gene therapies treat or prevent disease by tweaking the content or expression of cells' DNA.

LiveScience

'Miracle cure' for spinal disorder rolled out on NHS - but babies still face paralysis

A cure for a deadly spinal disorder will be given out on the NHS after pioneering gene therapy costing £1.8million was approved today for infants before symptoms begin

Daily Mirror

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'Gene therapy found to wind hearts BACK 10 years and protect people from ageing'

Scientists in Bristol have experimented with the therapy on mice and found it effectively rewound their hearts by the equivalent of 10 human years - soon they hope to use it on heart failure patients

Daily Mirror

Heartbreak as one sibling saved using miracle drug, but older sister not eligible for treatment

Teddi Shaw suffers from deadly metachromatic leukodystrophy and had her stem cells removed and the faulty genes replaced

The Independent UK

Little girl's life saved in Manchester by new gene therapy treatment in UK first

'Without this treatment, we would be facing both our children being taken away'

Manchester Evening News

Shilbottle toddler becomes first child in UK to receive revolutionary gene therapy for fatal disorder

Teddi Shaw, from Shilbottle, suffers from metachromatic leukodystrophy but has been given the chance of a normal life

Chronicle Live

Toddler Teddi is first child in UK to receive revolutionary gene therapy

Young Teddi Shaw will have a normal life thanks to one of the world’s most expensive drugs, but sadly her older sister was too far advanced in her illness to benefit from the treatment

Wales Online

Toddler becomes first child in UK to receive revolutionary gene therapy

Teddi Shaw suffers from deadly metachromatic leukodystrophy (MLD) but has been given the chance of a normal life.

The Independent UK

Girl with deadly inherited condition is cured with gene therapy on NHS

Teddi Shaw, from Northumberland, first recipient on health service of Libmeldy, world’s most expensive drug

The Guardian - UK

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Daily Mirror

'Miracle' gene therapy treatment cures baby facing death - but comes too late for sister

Little Teddi Shaw has become the first NHS patient to receive the treatment for metachromatic leukodystrophy. The 19-month-old was diagnosed with MLD last April

The Independent UK

Promising gene therapy delivers treatment directly to brain

Scientists say delivering gene therapy directly to the brain holds great promise

The Independent UK

NOT REAL NEWS: A look at what didn't happen this week

Social media users shared a range of false claims this week

Dynamite News

Health: Finds New Infant Gene Therapy To Treat Artemis-SCID Patients

A novel gene therapy developed at UC San Francisco is on track to improve the health of ten young children who were born without functioning immune systems and without the capacity to fight infections. Read on for details:

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Reuters

Sickle cell cure will be cost-effective if health disparities considered -study finds

(This Dec.13 story has been corrected to remove reference to Sanofi in the fifth paragraph)

The Guardian - UK

Revolutionary gene therapy offers hope for untreatable cancers

UK teenager is first person in the world to receive ‘base-edited’ cells that could transform cancer medicine

Tribune News Service

World’s most expensive drug approved to treat hemophilia at $3.5 million a dose

U.S. regulators approved CSL Behring’s hemophilia B gene therapy, a one-off infusion that frees patients from regular treatments but costs $3.5 million a dose, making it the most expensive medicine in the world.

The Guardian - US

Gene therapy at $3.5m a dose approved for US adults with hemophilia B

Hemgenix frees patients from repeated treatments but initial cost makes it most expensive medicine in the world

The Independent UK

$3.5M gene therapy for hemophilia gets FDA approval

U.S. health regulators have approved the first gene therapy for hemophilia, a blood-clotting disorder with few treatment options

The Conversation

Epilepsy: gene therapy technique targeting overactive brain cells shows promise in treating drug-resistant form of the condition

Something like 50 million people worldwide have epilepsy. While the majority of these people are able to use medications to manage and prevent their seizures, around one-third don’t respond well to these treatments. In such cases, the only option available to bring seizures under control is to remove the part…

The Independent UK

Death in CRISPR gene therapy study sparks search for answers

The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now trying to figure out what killed him

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