‘It would be huge to see my granddaughter’: In an attempt to cure blindness, scientists use CRISPR to edit DNA inside patients' bodies

‘It would be huge to see my granddaughter’: In an attempt to cure blindness, scientists use CRISPR to edit DNA inside patients’ bodies

Carlene Knight would love to do things that most people take for granted, such as read books, drive a car, ...
‘Optogenetics’ miracle? Gene therapy and high-tech goggles partially restore sight to man blinded for 40 years

‘Optogenetics’ miracle? Gene therapy and high-tech goggles partially restore sight to man blinded for 40 years

[A new gene therapy to treat blindness] relies on something called optogenetics. The idea is to edit nerve cells collected ...
Is science finally getting gene therapy right in the effort to fight intractable diseases?

Is science finally getting gene therapy right in the effort to fight intractable diseases?

Over time, researchers have devised a number of ways to manipulate the genetic activity within cells, often by giving them ...
Infographic: Gene therapy drugs that silence the effects of faulty genes could help tackle Huntington’s and other neurodegenerative diseases

Infographic: Gene therapy drugs that silence the effects of faulty genes could help tackle Huntington’s and other neurodegenerative diseases

Huntington’s disease (HD) is an inherited condition that causes widespread deterioration in the brain and disrupts thinking, behaviour, emotion and ...
A golden age of gene therapy is now in sight

A golden age of gene therapy is now in sight

To date, just two in vivo gene therapies have been approved in the United States. Both are for what [Duke ...
New hope for Duchenne muscular dystrophy (DMD) patients? CRISPR cured a mouse — and humans could be next

New hope for Duchenne muscular dystrophy (DMD) patients? CRISPR cured a mouse — and humans could be next

DMD is a fatal — and currently incurable — genetic condition that causes the body's muscles to deteriorate over time.  ...
Cure for sickle cell diseases inches closer with launch of gene therapy trial

Cure for sickle cell diseases inches closer with launch of gene therapy trial

For 65 years, scientists have known the cause of sickle cell disease but have been unable to cure it without ...
Gene transfer therapies are the ‘next big thing’ in medicine. Here’s how gene editing works and the companies behind it

Gene transfer therapies are the ‘next big thing’ in medicine. Here’s how gene editing works and the companies behind it

The breakthroughs made possible by gene editing were shown in the Jan. 6 news that base editing had repaired a ...
Delay aging and extend our lifespans? Gene therapy might be able to do that

Delay aging and extend our lifespans? Gene therapy might be able to do that

How many aging-promoting genes are there in the human genome? What are the molecular mechanisms by which these genes regulate ...
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30+ genes identified that could protect us from COVID, opening door to gene therapy prevention solutions

The goal was two-fold: to identify the genes that make human cells more resistant to SARS-CoV-2 virus; and test existing ...
Gene therapy may be able to restore vision

Gene therapy may be able to restore vision

In a normal eye, opsins are expressed by the rod and cone photoreceptors in the retina. When activated by light, ...
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Resurgence of gene therapy has dramatically altered the the biomedicine revolution

Some technologies that have emerged and altered the landscape in recent years include immunotherapy, CRISPR-Cas9 gene editing, and chimeric antigen ...
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‘Gigantic leaps forward’ What’s happened in the 21 years since a teenager died undergoing gene therapy, stalling emergence of this biomedical breakthrough?

In 1999, [teenager] Jesse [Gelsinger] received a dose of the ornithine transcarbamylase gene, engineered into a recombinant adenovirus, at the ...
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Podcast: Rare genetic disorders and pregnancy—Navigating an ’emotionally challenging’ journey

We look at the progress that’s been made in tackling rare genetic disorders (and the challenges that remain) and we ...
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Herpes cure? Gene therapy may work

Infectious disease researchers at Fred Hutchinson Cancer Research Center have used a gene editing approach to remove latent herpes simplex ...
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Gene therapy for hemophilia delayed until 2022 after FDA rejects one-time treatment, shocking doctors and scientists

U.S. regulators rejected [Biomarin’s] potentially game-changing hemophilia A gene therapy over concerns it might not really be a one-and-done lifetime ...
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One year in, 9 boys with muscular dystrophy show remarkable progress from gene therapy

Nine boys aged 6 to 12 who have been living with [Duchenne Muscular Dystrophy] since birth received a gene therapy ...
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Strokes often deadly but gene therapy offers hope to rebuild critically damaged brain cells

When you think of a typical meal in the US South, you will likely recall the rich, hearty dishes that ...
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Lucy may not live to see her second birthday without expensive gene therapy

Lucy Van Doormaal was born on April 1 in Vancouver, but before her parents, Scott and Laura Van Doormaal, could ...
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Another COVID-19 casualty: ‘Major delays’ for gene therapy trials for rare diseases

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has ...
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Gene therapy could restore color vision for people who see the world in shades of gray

In a small trial in Germany, an experimental gene therapy improved the vision of nine people with total color blindness, ...
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Ultimate fitness hack: Imagine being able to build muscles with a gene therapy

Trying to hack fitness is a multi-million-dollar industry; we’ve all seen at least one ad featuring a purported miracle product ...
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What’s holding up gene therapy? Making drugs on a large scale

As interest in gene therapy grows, manufacturers face physical, biological, and engineering challenges to developing and producing drugs on a ...
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Podcast: Fighting blindness with CRISPR. Ophthalmologist in groundbreaking study explains how gene editing could treat a once-incurable disease

Congenital eye disorders can rob children of their eyesight at a young age and severely diminish their quality of life ...
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Common form of inherited blindness shows ‘massive improvements’ with experimental gene therapy

This month, K.L. became one of the first patients to receive a new experimental gene therapy for children with a ...
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Building ‘better’ astronauts through genetic engineering could be key to colonizing other planets

Through genetic engineering, we will one day have the ability to thrive in harsh alien environments ...
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Gene therapy shows success against some cancers and inherited disorders. Can it tackle obesity?

Scientists are working with treatments that have shown success in amping up the metabolism of mice, helping them lose weight ...
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