Medical research

Researchers develop 'dimmer switch' to help control gene therapy

In a major advancement in the field of gene therapy for rare and devastating diseases, researchers at Children's Hospital of Philadelphia (CHOP) have developed a "dimmer switch" system that can control levels of proteins ...

Genetics

Cellular benefits of gene therapy seen decades after treatment

An international collaboration between Great Ormond Street Hospital, the UCL GOS Institute for Child Health and Harvard Medical School has shown that the beneficial effects of gene therapy can be seen decades after the transplanted ...

Medical research

Discovery challenges the foundations of gene therapy

A new publication by scientists from Children's Medical Research Institute has challenged one of the foundations of the gene therapy field and will help to improve strategies for treating serious genetic disorders of the ...

Neuroscience

Economic, fast and efficient cell-type targeting

It is a remarkable proof for the concept of IOB: working closely hand in hand, our molecular and clinical researchers have developed a library of 230 adeno associated viral vectors (AAVs), each with a different synthetic ...

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