Project will help blind children to see

By Cai Wenjun  |   2008-7-5  |     NEWSPAPER EDITION

EXPERTS from the United States are to take part in a project with local medical staff to help children blind from birth due to the mutation of a gene called RPE65.

Dr Zhao Peiquan, from Xinhua Hospital, who hosted an international forum on retinal disease in the city yesterday, said scientists had identified 14 genes with mutations that can cause Leber's Congenital Amaurosis, an inherited retinal degenerative disease characterized by severe loss of vision at birth, and clinical trials of gene replacement therapy had begun.

"It is such a breakthrough that the US launched the world's first gene therapy by injecting synthesized RPE65 into a blind person's eyes on October 25 last year," said Zhao. "So far, nine people in the US and UK have undergone the gene replacement therapy and all reported good results." He said the gene therapy was based a 30-year-old lab research and animal experiments. China was lagging far behind on gene synthesis and clinical practice.

"We have invited US experts to visit our hospital in September and participate a project to jointly detect and treat children with RPE65-related blindness," Zhao said. "Clinical diagnosis has found a dozen such children and a screening at Shanghai Blind Children's School will be carried out to find suitable patients."

According to medical experts, gene therapy is the most promising treatment for optical diseases.