Condition or Therapy: X-linked severe combined immunodeficiency (SCID-X1); gene therapy
Category: Immunology

What is the goal of this study?

Researchers at Seattle Children’s are testing whether gene therapy can be used to treat male infants with X-linked Severe Combined Immunodeficiency (X-linked SCID).

Who can join the study?

This study might be a good fit if your child:

  • Has X-linked SCID
  • Is over the age of 2 months 
  • Does not have a matched sibling donor

What happens in the study?

If you decide to have your child take part in this study and your child is a good candidate for this trial, your child would:

  • Undergo a bone marrow cell harvest
  • Receive a reduced amount of chemotherapy to create space for gene modified cells to grow
  • Receive his own gene modified cells
  • Have regular evaluations to monitor response to therapy

Who can I contact for information or to enroll?

You can email or call the study team at 206-884-1068.


Study Location(s): Seattle Children's main campus
Principal Investigator: Dr. Aleksandra Petrovic