gene therapy

Yesterday, I had the pleasure of traveling to Washington, DC to film on set at Al Jazeera for a live television program that engaged a global discussion on sickle cell anemia, its perils and the advances in gene therapy that are showing great promise for this genetic disease. 

The host of the program is Femi Oke and the digital co-host is Malika Bilal (pictured with me). Questions were fielded from around the world utilizing various social media platforms as the show was not only streamed live on The Stream and Al Jazeera English television, but is re-aired on Al Jazeera media networks worldwide.

You can check it out above...

A number of recent headlines imply a case study just published in the New England Journal of Medicine proves that gene therapy has cured sickle cell disease—a genetic disorder that incurs tremendous pain, suffering and diminished life expectancy. Here, we will unpack the significance of the researchers’ findings.

First, let’s address why this news could be so groundbreaking to those afflicted and their loved ones.

Sickle Cell Disease is an inherited condition that causes a mutated hemoglobin—the protein within red blood cells (RBCs) that carries oxygen for delivery to vital tissues. Oxygen feeds our organs so they can stay healthy and perform their respective jobs. This Hemoglobin S (aka Sickle...

gene therapy via shutterstock gene therapy via shutterstock

Hemophilia was once popularly referred to as the royal disease. It was passed through the blood line of Queen Victoria to her progeny, which affected several royal families in Spain, Germany and Russia.

Hemophilia is an inherited bleeding disorder that can be either X-linked, meaning passed along on the X chromosome of female carriers primarily affecting male offspring, since males inherit only one copy of the X chromosome containing the defective gene. Or they can be...

Altered DNA in Duchenne MDIn an amazing, salutary coincidence, three independent research groups recently reported progress utilizing gene modification approaches to reducing the muscular dysfunction characteristic of sex-linked Duchenne-type Muscular Dystrophy, the most common and severe form of the disease.

The current studies were done on mice, but in this instance mouse and human gene mutations are quite similar, both biochemically and pathophysiologically re: the muscular dysfunction resulting from the genetic aberration.

Duchenne muscular dystrophy (DMD) is the most common fatal genetic disorder...

elephant-1389977-640x480Some unfounded scientific beliefs tend to persist in popular culture for years, even decades, despite being sufficiently debunked by reputable sources.

If you were to watch CBS on Tuesday nights, you'd see a whole TV series (there's a movie, too) based on the thoroughly-debunked idea that humans only use 10 percent of their brain. Humans actually use all of their brain.

Another...

shiny-brain-1150907-1279x1065It took roughly 20 years from the time we realized that DNA was the source of genetic material -- proteins were considered, as well -- to decide we should start manipulating it in living humans. At the time the idea was novel, but rather simple: If a gene is broken, why not replace it with one that isn't?

Getting from rhetoric to execution has been difficult, as an effective...

1037197_16221105Scientists and doctors alike have considered gene therapy a potential panacea since it was first postulated on in the 1970s. If harnessed correctly, gene therapy could provide real cures for an array of ailments from cancers to Parkinson s to Tay-Sachs. The common thread in these is that a cell s genome is altered in a very specific way that is detrimental to the organism s physiology. In gene therapy, the idea is that by using a...

Brain MRI-Alzheimer'sParkinson s disease is the second most common neurodegenerative disorder after Alzheimer s, affecting about 5 million people worldwide, including at least 50,000 new cases in the U.S. each year. The symptoms of Parkinson s are due to loss of dopamine-producing nerve cells in the part of the brain responsible for controlling movement.

The most widely used treatment is the drug levodopa, a precursor of dopamine that can cross the blood-brain barrier. However, over time, cell death is...