Researchers analysed thousands of laboratory-made plasmids and discovered that nearly half of them had defects, raising questions of experimental reproducibility.
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A baby girl born with profound genetic deafness can now hear unaided after receiving a “groundbreaking” gene therapy trial, Britain’s National Health Service said Thursday.
Opal Sandy, an 18-month-old from Oxfordshire, England, is the first patient treated in a global gene therapy trial that is showing “mind-blowing” results, Addenbrooke’s Hospital in Cambridge said in a statement. Opal is “the first British patient in the world and the youngest child to receive this type of treatment,” the hospital said.
The treatment is already having practical results, the hospital said: Opal can now respond to her parents’ voices and can communicate words such as “Dada” and “bye-bye.”
“When Opal could first hear us clapping unaided it was mind-blowing,” her mother, Jo Sandy, said in a statement. “We were so happy when the clinical team confirmed at 24 weeks that her hearing was also picking up softer sounds and speech.”
Her father, James Sandy, said that it was “already making a difference to our day-to-day lives, like at bath-time or swimming,” he said. “… We feel so...