Gene therapy delivered directly to the brain treats extremely rare disease in children

Gene therapy delivered directly to the brain treats extremely rare disease in children

Laura Ungar | 
When Rylae-Ann Poulin was a year old, she didn’t crawl or babble like other kids her age. A rare genetic ...
‘Garage scientists’ can biohack the latest health craze, but also a bioweapon. Should we be worried?

‘Garage scientists’ can biohack their way to the newest health craze — and potentially make bioweapons. Should we be worried?

Lauren Richards | 
Anyone who can follow a recipe and has enough money to buy a pipette can modify their DNA at home ...
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‘Perfect human’? Genetics researchers ponder ethical consequences of tinkering with human DNA

Robin McKie | 
Researchers are investigating ways to use genome editing to tackle sickle cell anaemia, muscular dystrophy, cancer, diabetes and some forms ...
Here is a new AI-powered biotech innovation that could offer unique advantages over CRISPR when gene-editing disease therapies

Zinc fingers: New AI-powered biotech innovation could offer unique advantages over CRISPR when building gene-editing disease therapies

Christopher McFadden | 
A new study has developed what the researchers call the "world's first" simple, modifiable proteins. Called "zinc fingers," these special ...
CRISPR gene editing can treat heart disease and repair damaged tissue after a heart attack

CRISPR gene editing can treat heart disease and repair damaged tissue after a heart attack

Corrie Pelc | 
Every year cardiovascular disease accounts for about 32% of all deaths worldwide. Researchers from the University of Texas Southwestern Medical ...
Viewpoint: Malaria kills half a million people each year. Here’s how CRISPR gene edited mosquitoes could end this scourge

Viewpoint: Malaria kills half a million people each year. Here’s how CRISPR gene edited mosquitoes could end this scourge

Dora Wells | 
Genetically modified mosquitoes … could CRISPR gene editing end malaria? This article tells the story of Target Malaria, a project ...
Cancer vaccine progress: CRISPR-developed shot stops brain tumors in mice

Cancer vaccine progress: CRISPR-developed shot stops brain tumors in mice

Cami Rosso | 
Cancer of the brain and nervous system accounted for over 250,000 deaths worldwide in 2020 ...
Could a vaccine permanently protect us from heart disease? CRISPR gene editing poised to provide healthcare solutions for most everyone

Could a vaccine permanently protect us from heart disease? CRISPR gene editing poised to provide healthcare solutions for most everyone

Jessica Hamzelou | 
Last year, Verve Therapeutics started the first human trial of a CRISPR treatment that could benefit most people—a signal that ...
Podcast: Viewpoint — ‘Bigotry dressed up as biology: Geneticist Adam Rutherford’s 8 worrisome trends

Viewpoint — ‘Bigotry dressed up as biology’: Geneticist Adam Rutherford’s 8 surprising facts about eugenics

Adam Rutherford, Lisa Godfrey, Nahlah Ayed | 
Adam Rutherford is a geneticist and the author of Control: The Dark History and Troubling Present of Eugenics where he ...
CRISPR gene editing scientist who illegally manipulated embryos wants to cure genetic diseases. Should the scientific community trust him?

CRISPR gene editing scientist who illegally manipulated embryos wants to cure genetic diseases. Should the scientific community trust him?

Emily Mullin | 
He Jiankui discusses his plans for finding cures for devastating genetic diseases. Should the scientific community trust him? ...
CRISPR gene editing is in clinical trials to treat sickle cell disease, cancer, HIV/AIDS and some rare inherited diseases. Here’s what we can expect going forward

CRISPR gene editing is in clinical trials to treat sickle cell disease, cancer, HIV/AIDS and some rare inherited diseases. Here’s what we can expect going forward

Brian Owens | 
When Jennifer Doudna and Emmanuelle Charpentier revealed that the bacterial Crispr–Cas9 antiviral defence system could be reprogrammed to edit genomic ...
Have gene therapy and gene editing techology progressed enough to deliver life-saving results?

Have gene therapy and gene editing techology progressed enough to deliver life-saving results?

Doug Johnson | 
Gene therapy has had a long and bumpy history. Although researchers have made some notable and recent progress, past failures—including some ...
Using cancer cells to kill tumors? Cell therapy engineered to eliminate and prevent brain cancer in advanced mouse model

Using cancer cells to kill tumors? Cell therapy engineered to eliminate and prevent brain cancer in advanced mouse model

Researchers took advantage of living tumor cells' unique ability to travel long distances across the brain to return to the ...
‘Zero detectable cancer cells’: Experimental CRISPR tool appears to cure 13-year-old’s ‘incurable’ leukemia

‘Zero detectable cancer cells’: Experimental CRISPR tool appears to cure 13-year-old’s ‘incurable’ leukemia

Deena Theresa | 
World-first: Experimental CRISPR technology removes detectable cancer cells in 13-year-old. Alyssa received genetically modified cells that could chase and kill ...
Single shot genetic cures are coming. Will insurance companies pay for these multi-million dollar therapies?

Single shot genetic cures are coming. Will insurance companies pay for these multi-million dollar therapies?

Emily Mullin | 
Some of Steven Pipe’s hemophilia patients consider themselves cured. In a trial Pipe led from 2018 to 2021, they received ...
Can CRISPR gene editing be used to decimate populations?

CRISPR and gene drives: Revolutionary tool to address pest control or potential weapon of mass destruction?

Jimmy Ng | 
You’ve probably heard of the gene-editing tool CRISPR, often referred to as “word processing for DNA.” Much of the attention ...
CRISPR

CRISPR gene editing could help cure as many as 7,000 diseases. What’s holding things up?

Fyodor Urnov | 
The parents of a 2-year-old girl write that their daughter “could die within the next year” because a genetic mutation ...
He Jiankui released after 3 years in prison

He Jiankui — CRISPR babies mastermind — is out of prison. What’s next for his career?

Megan Molteni | 
He Jiankui, the Chinese biophysicist who created the first gene-edited children, had been quiet since completing a three-year prison sentence in April, ...
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Single-dose CRISPR gene editing technology helps train patients’ immune systems to fight cancer

Beth JoJack | 
For the first time, researchers have used CRISPR technology to substitute genes in patients’ immune cells to treat cancer. Participants ...
How CRISPR could help lower LDL

Poor cholesterol? A gene-edited solution could lower ‘bad’ LDL and reduce heart disease risk

Markham Heid | 
While a poor diet, stress, lack of exercise, and other lifestyle factors can lead to cholesterol problems, genetic factors also ...
Children born of the great depression

Children born during the great depression aged rapidly later in life. Here’s how trauma can affect you before you’re even born

Freda Kreier | 
The worst recession in US history shaped how well people would age — before they were even born. Researchers have ...
Viewpoint: Gene editing is revolutionizing medicine — but it opens a Pandora’s box of ethical questions

Viewpoint: Gene editing is revolutionizing medicine — but it opens a Pandora’s box of ethical questions

Hilary Sheppard | 
Genetic engineering or genetic modification has long been a headline grabber, often framed in a negative light. However we need ...
Viewpoint: ‘What’s the biological equivalent of an atomic bomb’? Here’s how the gene editing revolution could go astray

Viewpoint: ‘What’s the biological equivalent of an atomic bomb’? Here’s how the gene editing revolution could go astray

Matthew Herper | 
We now sequence the genes of people, animals, plants and tumors routinely. We’re starting to edit DNA, not only in ...
CRISPR co-creator Jennifer Doudna looks back on the best and worst parts of gene editing’s first ten years

CRISPR co-creator Jennifer Doudna looks back on the best and worst parts of gene editing’s first ten years

Jennifer Doudna, Julianna LeMieux | 
In a candid conversation with GEN Senior Science Writer Julianna LeMieux during “The State of Biotech” forum, Doudna discussed a ...
‘We are out on the far edge of experimentation’: Lone volunteer in Duchenne muscular dystrophy CRISPR gene editing study dies

‘We are out on the far edge of experimentation’: Lone volunteer in Duchenne muscular dystrophy CRISPR gene editing study dies

Laura Ungar | 
The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now ...
Gene therapy update: More than 2,000 treatments are in development worldwide as revolution spreads to low- and middle-income countries

Gene therapy update: More than 2,000 treatments are in development worldwide as revolution spreads to low- and middle-income countries

Sanjeev Khagram, Shyam Bishen | 
Gene therapy is at the forefront of modern medicine. By making precise changes to the human genome, these sophisticated technologies ...
ban on gene-editing of heritable genomes

Drawing a line: European Convention upholds ban on heritable human genome editing

Pete Shanks | 
The Council of Europe’s Convention on Human Rights and Biomedicine, better known as the Ovideo Convention, explicitly bans heritable human ...
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