Thursday December 13, 2018

Gene Therapy Wins Big at Portugal’s Champalimaud Foundation

The foundation, which focuses on neuroscience and oncology research at its Lisbon base, was set up at the bequest of Portugal's late industrialist Antonio Champalimaud.

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Gene Theory
ean Bennett, Albert Maguire, Robin Ali, James Bainbridge, Samuel Jacobson, T. Michael Redmond and Portugal's President Marcelo Rebelo de Sousa attend the 2018 Antonio Champalimaud Vision Awards ceremony at Champalimaud Foundation in Lisbon, Portugal, VOA
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Seven scientists in the United States and Britain who have come up with a revolutionary gene therapy cure for a rare genetic form of childhood blindness won a 1 million euro ($1.15 million) prize Tuesday, Portugal’s Champalimaud Foundation said.

Established in 2006, the annual award for work related to vision is one of the world’s largest science prizes, more than the latest 9 million Swedish crown ($987,000) Nobel Prize in Physiology or Medicine.

“This is the first, and still only, example of successful gene therapy in humans that corrects an inherited genetic defect and is therefore a milestone in medical therapeutics,” said Alfred Sommer, Dean Emeritus of the Johns Hopkins Bloomberg School of Public Health and chairman of the award jury.

Gene
Colour sensitivity can also led to Retinal Diseases/Blindness. Pixabay

One of those honored, Michael Redmond of the National Eye Institute in Maryland, had traced the cause of the disease, Leber congenital amaurosis (LCA), to a mutated gene.

Three cooperating research teams later managed to replace the gene in the eye, restoring vision to treated children and adults with one form of LCA and “enabling the entire field of gene therapy for human disease,” the foundation said.

These teams are comprised of U.S. scientists Jean Bennett and Albert Maguire; Samuel Jacobson and William Hauswirth; and Britons Robin Ali and James Bainbridge.

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Their gene augmentation therapy involved the delivery of healthy genes using engineered harmless viruses, described by the foundation as “an elegant solution.”

The foundation, which focuses on neuroscience and oncology research at its Lisbon base, was set up at the bequest of Portugal’s late industrialist Antonio Champalimaud who died in 2004. The first vision prize was awarded in 2006. (VOA)

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New Drug Offers Treatment For Diabetes-Related Blindness

The researchers now plan to conduct a full-scale clinical trial, Gamble said

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New drug offers hope for diabetes-related blindness.

In a major breakthrough, Australian scientists have developed a new drug that offers treatment for people suffering from diabetic retinopathy — the main cause of blindness from diabetes.

The debilitating disease occurs when tiny blood vessels in the retina, responsible for detecting light, leak fluid or haemorrhage.

While treatment options include laser surgery or eye injections of anti-vascular endothelial growth factor (VEGF), they are not always effective or can result in side effects, highlighting the need for alternative therapeutic approaches.

The team from the Centenary Institute in Sydney developed a novel drug CD5-2, which in mouse models was found to mend the damaged blood retinal barrier and reduce vascular leakage.

“We believe CD5-2 could potentially be used as a stand-alone therapy to treat those patients who fail to respond to the anti-VEGF treatment. It may also work in conjunction with existing anti-VEGF treatments to extend the effectiveness of the treatment,” said lead author Ka Ka Ting from the Institute.

“With limited treatment options currently available, it is critical we develop alternative strategies for the treatment of this outcome of diabetes,” Ting added.

Diabetes
Representational image. Pixabay

The key process involved in diabetic retinopathy pathology is the breakdown of the blood-retinal barrier (BRB), which is normally impermeable. Its integrity relies on how well capillary endothelial cells are bound together by tight junctions. If the junctions are loose or damaged, the blood vessels can leak.

In the study, reported in the journal Diabetologia, CD5-2 was found to have therapeutic potential for individuals with vascular-leak-associated retinal diseases based on its ease of delivery and its ability to reverse vascular dysfunction as well as inflammatory aspects in animal models of retinopathy.

Previous studies have shown that CD5-2 can have positive effects on the growth of blood vessels.

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“This drug has shown great promise for the treatment of several major health problems, in the eye and in the brain,” said Professor Jenny Gamble, head of Centenary’s Vascular Biology Programme.

The researchers now plan to conduct a full-scale clinical trial, Gamble said. (IANS)