What rare genetic condition did the trial aim to treat?

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The trial aimed to treat a rare genetic condition causing cognitive development delay.

An experimental gene therapy trial was conducted with the aim of treating a rare genetic condition that resulted in delayed cognitive development. The therapy involved a spinal infusion of trillions of viruses designed to penetrate the brain.
The Specific Condition Remains Undisclosed

The precise name of the rare genetic condition targeted by the experimental therapy has not been publicly disclosed in the available information. The report indicates that the treatment was intended to address a condition leading to a delay in cognitive development.
Background of Gene Editing Research

Globally, gene editing research aims to correct genetic defects. In this specific case, the lead researcher, neuroscientist Zilong Qiu, was reportedly seeking to develop the world's first gene-editing therapy directed at the brain. The objective was to rewrite a mutated gene in the patient's neurons to enable the production of a vital protein.
Financial Investment and Ethical Concerns

The experiment, which utilized a pseudonym "Mei" for the six-year-old girl, involved a significant financial investment from her parents, exceeding US$800,000. Reports suggest that Mei's parents were not adequately informed about the potential risks associated with the therapy. The university has initiated a comprehensive investigation into the incident, emphasizing its commitment to scientific research integrity and ethical research norms.

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What rare genetic condition did the trial aim to treat?

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