Skip to main content
Premium Trial:

Request an Annual Quote

Gene Therapy for SCID 'Encouraging'

A gene therapy appears to be effective in treating severe combined immunodeficiency syndrome, or SCID, the Associated Press reports.

Researchers from the University of California, Los Angeles, and Great Ormond Street Hospital treated 50 children with SCID due to adenosine deaminase deficiency. ADA-SCID is caused by mutations in the ADA gene, which leads to an immune system that does not function properly and can often be fatal. As they report in the New England Journal of Medicine, the researchers used lentiviral vector-based gene therapy to deliver a working version of the gene to the children in their study.

Overall, 48 of the 50 children had restored ADA enzyme and immune system function following treatment and through 24 to 36 months of follow up. "They're no longer patients. They're off all of their antibiotics. They're living normal lives," senior author Donald Kohn from UCLA tells HealthDay.

The AP notes that it not yet clear how long the effect will last, but Stephen Gottschalk of St. Jude Children's Research Hospital, who was not involved in the study, tells it that the "immune function seems stable over time so I think it looks very, very encouraging."

The Scan

Vaccine Update Recommended

A US Food and Drug Administration panel recommends booster vaccines be updated to target Omicron, CNBC reports.

US to Make More Vaccines for Monkeypox Available

The US is to make nearly 300,000 vaccine doses available in the coming weeks to stem the spread of human monkeypox virus, according to NPR.

Sentence Appealed

The Associated Press reports that Swedish prosecutors are appealing the sentence given to a surgeon once lauded for transplanting synthetic tracheas but then convicted of causing bodily harm.

Genome Biology Papers on COVID-19 Effector Genes, Virtual ChIP-seq, scDART

In Genome Biology this week: proposed COVID-19 effector genes, method to predict transcription factor binding patterns, and more.