Gene Therapy
Gene therapy creates boy’s replacement skin from his stem cells
Doctors treated a 7-year-old boy’s devastating genetic skin disease—junctional epidermolysis bullosa—by genetically modifying his stem cells to create a new ...
What’s the excitement over gene therapy?
Gene therapy has been in the news a lot of late as a potential cure for a variety of genetic ...
Fighting malaria: Bacteria targets parasites in mosquito gut
Using a bacteria strain that colonizes the mosquito digestive tract and spreads rapidly throughout the populations, scientists have successfully inhibited ...
Safer CRISPR? Cas13 version edits RNA, not DNA
CRISPR, while a major leap forward in gene editing, can still be a blunt instrument. There have been problems with ...
Biohackers help ‘patient’ inject himself with experimental HIV treatment—live on Facebook
[Tristan] Roberts is about to inject himself with an experimental gene therapy for HIV, a DIY prototype treatment designed by ...
Protecting against cancer: What can we learn from animals who live for centuries
There are an increasing number of genetic clues from animals that could provide hints to treating aging and age related ...
Gene therapy that reboots body’s immune cells to fight non-Hodgkin’s lymphoma approved by FDA
The Food and Drug Administration on [October 19] approved the second in a radically new class of treatments that genetically reboot ...
‘Synthetic lethality’ targets cancer cells by damaging vulnerable DNA
By designing small molecules that can “turn off” kinase enzymes stuck in the “on” position, they have managed to ward ...
Should the European Union relax GMO clinical trial guidelines?
Four organisations representing pharmaceutical firms, biotech companies, universities, and research institutes are calling on the European commission to update, streamline ...
CRISPR ear injections repair genetic hearing loss in mice
We all know that CRISPR is the next big thing in gene-editing treatments. But how do you get the versatile genetic scissors ...
Edible ‘CRISPR pill’ could make harmful bacteria self-destruct
As resistance to antibiotics grows in the U.S., researchers are looking for new ways to fight germs like Clostridium difficile, a ...
Upchuck factor: Alcoholism targeted with novel gene therapy
Researchers are exploring the use of a naturally occurring mutation that causes people to get sick after drinking alcohol. The ...
David Gorski: Rigvir ‘virotherapy’ another unproven alternative medicine cancer therapy
[Editor's note: Dr. David Gorski, MD, PhD, FACS is a surgical oncologist at the Barbara Ann Karmanos Cancer Institute.] [Cancer ...
First-ever gene-altering leukemia treatment approved by the FDA, uses patient cells to fight cancer
The Food and Drug Administration on Wednesday approved the first-ever treatment that genetically alters a patient’s own cells to fight cancer, a milestone ...
Muscular dystrophy ‘death sentence’ targeted by gene therapy trials
[T]hree U.S. teams say they are ready to try to treat Duchenne [Muscular Dystrophy] with gene therapy. The first study ...
‘Genome cloaking’ could protect genetic privacy in medical tests
It is now possible to scour complete human genomes for the presence of disease-associated genes without revealing any genetic information ...
RNA editing could help slow progression of symptoms of ALS and Huntington’s
The most common gene editing technique, CRISPR-Cas9, only modifies DNA. That's helpful in most cases, but it means that you can't ...
Could vitamin B3 supplements prevent birth defects?
An extra dose of vitamin B3 might help prevent certain kinds of complex birth defects, according to a new study ...
Three-person IVF as an infertility solution has a long way to go
The procedure is typically targeted at parents who want to avoid passing a potentially fatal mitochondrial disease to their child ...
Anti-aging hormone boosts cognitive abilities in mice – could humans be next?
[Klotho is] a naturally occurring hormone in the body. More than two decades ago, Japanese researchers discovered that this hormone ...
Could gene editing have saved Charlie Gard?
The British infant died of a mitochondrial disease as news broke that US scientists had successfully edited a human embryo ...
CRISPR revolution: How scientists are turning gene-editing hype into food and medical breakthroughs
The powerful gene-editing tool is becoming ubiquitous, appearing in the media with ever increasing frequency. But it's more than just ...
Why use gene editing on human embryos when other options are available?
Researchers in the U.S. and South Korea have shown that gene editing can correct a genetic disorder in an embryo. Using CRISPR-Cas9, ...
We each have 3 billion base pairs in our genome. Artificial intelligence can help us sort it out.
Genes carry the information that make you you. So it's fitting that, when sequenced and stored in a computer, your ...
Are obesity, diabetes and other diseases treatable using gene-therapy skin grafts?
A research team based at the University of Chicago has overcome challenges that have limited gene therapy and demonstrated how ...
Pioneering gene therapy restores vision to people with rare retinal blindness
Spark Therapeutics is moving forward with its treatment for a type of inherited retinal dystrophy. In the wake of the ...
Muscular dystrophy in dogs reversed with gene therapy–are children next?
Duchenne muscular dystrophy (DMD) is the most common and, sadly, the most severe form of the debilitating genetic disorder. Now ...