Tech & Sci
2019.02.19 16:22 GMT+8

Where are we now in gene therapy treatments?

Pan Zhaoyi

Using a treatment targeting age-related macular degeneration (AMD), Janet Osborne has become the first women in the world to have gene therapy which aims to stop blindness, according to the BBC.

AMD is the leading cause of severe vision loss in adults over age 50.

The therapy, carried out at Oxford Eye Hospital by Robert MacLaren, professor of ophthalmology at the University of Oxford, is expected to keep patients' macular healthy by injecting a harmless virus at the back of the eye.

Prof MacLaren explained: “The idea of this gene therapy is to ‘deactivate' the (immune system), but at a very specific point at the back of the eye, so the patient would otherwise be unaffected by it."

 A woman at an eye examination /VCG Photo

Gene therapy development blossoms worldwide

Breakthroughs are not limited in eye disorders treatment, scientists in the University of North Carolina used a gene-editing tool to treat a rare brain disorder in unborn babies. This marked a huge step in treating children with walking and sleeping disorders, as well as intellectual disabilities.

The lead researcher believes that if future work goes well, his therapy could be an important path that paves the way for treating other, more common disorders, such as certain forms of autism.

Studies show that gene therapy administered in the womb could be used to treat a deadly genetic disease, a discovery found in fetal mice.

Before that, no one has ever developed a gene therapy treatment that was required to be injected directly into the brains of a fetus.

Baby in foetus /VCG Photo

The U.S. Food and Drug Administration approved the first gene therapy for adults and children only in 2017.

While in China, except for the much-hyped and controversial gene-edited babies, Chinese scientists have been working on these gene-cut-and-paste researches for a long time.

Last August, a team of researchers at Kunming Institute of Zoology under the Chinese Academy of Sciences found the gene-editing tool could inhibit tumor growth by kicking out one key gene in the cells. 

But the experiment proved successful only in the lab, it's still not available for clinical trials.

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