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gene therapy

A new gene editing technique shows promise for the treatment of Duchenne muscular dystroph...

Researchers at the University of Texas (UT) Southwestern Medical Center have developed a technique that corrects a mutation leading to Duchenne muscular dystrophy (DMD). The technique, called CRISPR/Cas9-mediated genome editing, removes the mutation entirely in mice, and could have far-reaching consequences in the treatment of muscular dystrophy in people.  Read More

New technology allows the heart to become its own pacemaker (Image: Shutterstock)

Pacemakers serve an invaluable purpose, by electrically stimulating a recipient's heart in order to keep it beating at a steady rate. The implantation of a pacemaker is a major surgical procedure, however, plus its presence in the body can lead to complications such as infections. Now, for the first time, scientists have instead injected genes into the defective hearts of pigs, converting unspecialized heart cells into "biological pacemakers."  Read More

For the first time, researchers at the University of New South Wales in Australia have use...

According to the World Health Organization, more than 360 million people worldwide live with disabling hearing loss, and for many, devices such as hearing aids and cochlear implants allow them to maintain a normal life style. But what if a cochlear device could offer a biological solution that would enhance a patient’s experience?  Read More

The US Supreme Court has ruled that human genes cannot be patented Image: Shutterstock)

In what is being ballyhooed as a landmark decision likely to set the course of DNA-based diagnostic and therapeutic medicine for the next several decades, the US Supreme Court unanimously decided on June 13 that human genes are not patentable. Rather than objects invented or discovered, human genes are henceforth to be treated as "naturally occurring phenomena," and hence fail the patentability test under 35 USC 101. As is usual in patent cases, however, the ruling contains delicate shades of meaning.  Read More

Led by Fàtima Bosch (fifth from left), a University of Barcelona research team has cured d...

Researchers from the Universitat Autònoma de Barcelona (UAB) have claimed a first by successfully using a single session of gene therapy to cure dogs of type 1 diabetes. The work has shown that it is possible to cure the disease in large animals with a minimally-invasive procedure – potentially leading the way to further developments in studies for human treatment of the disease.  Read More

A new gene therapy approach could lead to the end of drug treatments for HIV patients (Ima...

One of the biggest problems in treating HIV patients is the amount of daily individual medications it takes to keep the virus at bay. In a new study, scientists at the Stanford University School of Medicine have engineered a new approach to tailored gene therapy that they say makes key cells of the immune system resistant to attack from the HIV virus, which may eventually lead to the removal of life-long dependencies on drugs for patients living with HIV.  Read More

Each nanochannel electroporation device incorporates two reservoirs joined by a nanoscale ...

One of the key processes in gene therapy involves taking cells from the patient, injecting a therapeutic genetic material into them, then reintroducing them to the patient’s body and letting them go to work. Unfortunately, getting that material into the cells can be tricky. While larger cells can actually be punctured with a fine needle, most human cells are too small for that approach to be possible. There are also methods of inserting random amounts of material into bulk quantities of cells, but these are inexact. Now, however, scientists at Ohio State University are reporting success with a process known as “nanochannel electroporation” (NEP), in which therapeutic biomolecules are electrically shot into cells.  Read More

Scientists are experimenting with using genetically engineered spider silk proteins in the...

Spider silk is pretty amazing stuff. Pound for pound, it has a tensile strength close to that of steel while being one-fifth as dense, it’s tougher than Kevlar, and it can stretch to almost one-and-a-half times its length without breaking. As if that wasn’t enough, it now appears that a genetically engineered version of the substance could be used for delivering genes into human cells.  Read More

Micrograph showing prostatic acinar adenocarcinoma - the most common form of prostate canc...

Using a virus containing a ‘library’ of DNA, researchers from the University of Leeds in the U.K., working with the Mayo Clinic in the U.S., have developed a vaccine that was able to destroy prostate cancer tumors in mice, while leaving healthy tissue untouched. Because the virus contains multiple fragments of genes, the vaccine is able to produce many possible antigens thereby boosting its effectiveness. The technique could be used to create vaccines to treat a wide range of cancers, including breast, pancreatic and lung tumors.  Read More

Scientists have used Salmonella bacteria (pictured) to eliminate viruses in mice (Photo: V...

Generally speaking, it is inadvisable to eat foods containing Salmonella bacteria – especially if you’re not a fan of diarrhea, fever or abdominal cramps. In the future, however, we might be swallowing genetically-engineered versions of the little guys as a way of treating viral infections. If we do, it will be thanks to research presently being carried out at UC Berkeley’s School of Public Health. Scientists there have reprogrammed Salmonella bacteria to act as harmless transporters of virus-stopping enzymes.  Read More

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