Eliza Gets Her Gene Therapy

ICYMI - Patient #1 Dosed in Pioneering #GeneTherapy Trial  #SNRTG #BigData #RareDisease @PLOS

  • The author of several textbooks and thousands of articles in scientific, medical, and consumer publications, Ricki’s first narrative nonfiction book, “The Forever Fix: Gene Therapy and the Boy Who Saved It,” was published by St. Martin’s Press in March 2012.
  • The gene therapy, at Nationwide Children’s Hospital in Columbus, Ohio, introduces the gene aboard an adeno-associated virus (AAV9) vector as a one-time intravenous injection.
  • Sanfilippo syndrome is a lysosomal storage disease (LSD).
  • “MPS IIIA is a deadly lysosomal storage disease with no approved treatments.
  • Eliza Gets Her Gene Therapy

Read the full article, click here.


@Primary_Immune1: “ICYMI – Patient #1 Dosed in Pioneering #GeneTherapy Trial #SNRTG #BigData #RareDisease @PLOS”


Keeping a little girl at home for 722 days may seem cruel, but for the O’Neill family, it was a demonstration of astonishing bravery and love.


Eliza Gets Her Gene Therapy

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