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Normal IgH Repertoire Diversity in an Infant with ADA Deficiency After Gene Therapy.

Carolyn H BalohSamiksha A BorkarKai-Fen ChangJiqiang YaoMichael S HershfieldSuhag H ParikhDonald B KohnMaureen M GoodenowJohn W SleasmanLi Yin
Published in: Journal of clinical immunology (2021)
Based on deep sequencing, gene therapy resulted in an IgHV repertoire with molecular diversity similar to healthy infants.
Keyphrases
  • gene therapy
  • high throughput sequencing
  • single cell
  • single molecule
  • replacement therapy