Nature is investigating concerns surrounding a paper at the heart of a news story published last week on the death of a 6-year-old girl following a gene therapy treatment in China.
The paper, published in February by a team led by neuroscientist Zilong Qiu of Shanghai Jiao Tong University School of Medicine in China, described preclinical work for a gene-editing treatment for Snijders Blok-Campeau syndrome, a neurodevelopmental disorder.
As we reported in collaboration with Science, the work was funded in part by the family of a 6-year-old girl with the condition. The family had approached Qiu based on work he had done with gene therapy for Rett syndrome, a disorder that also causes developmental delay. The parents ended up paying $860,000 to fund preclinical work for a gene-editing treatment for their daughter, who died of a severe immune reaction to the therapy seven days after receiving it.
The paper did not disclose the girl’s death or the family’s financial contribution, even though a preprint of the work did “thank the families with SNIBCPS patients for their participation and support in this study.”
In addition, several experts interviewed for the article questioned some of the study’s efficacy data.
Victoria Aranda, deputy editor of Nature, said no information about a planned trial in people was ever shared with the journal or mentioned in the submitted study. “The manuscript that was submitted to us … described preclinical feasibility work only — a mouse model and non-human primate experiments,” she told Retraction Watch by email. “We are now investigating concerns that have been raised around this manuscript and an Editors’ Note has been placed on the paper to reflect this.”
The editor’s note, dated July 29, states:
Readers are alerted that concerns have been raised regarding this article, including with the data presented. Further editorial action will be taken if appropriate as soon as the investigation into the concerns is complete and all parties have been given an opportunity to respond in full.
Qiu’s university has also launched an investigation, Science reported earlier this week. Shanghai Jiao Tong University School of Medicine said in a statement it would establish “a special task force to conduct a comprehensive investigation into the incident.”
Neither the university nor Xinhua Hospital, where the young girl was treated, responded to requests for comment prior to the publication of the story in Science.
The story has attracted international coverage by news outlets including the New York Post, Bloomberg, Times Higher Education, Southern Weekly and the South China Morning Post.
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