Severe combined immunodeficiency caused by mutations in the IL2RG gene on the x-chromosome (SCID-X1 or "bubble boy disease") is a devastating genetic disease that results in boys not being able…
Successful stem cell therapy requires the replacement of diseased or dysfunctional stem cells with healthy ones. These healthy stem cells can come from either a donor or can be stem…
This trial proposes to replace SCID patients' dysfunctional immune cells with healthy ones using a safer form of bone marrow transplant (BMT). Current BMT procedures must use toxic chemotherapy to…
St. Jude Children's Research Hospital is teaming up with UC San Francisco to repair the damaged immune system of children born with SCID. They will genetically modify the patient's own…
Therapeutic Candidate or Device CD34+CD90+ hematopoietic stem cells (HSC) in combination with AMG 191, a humanized anti-CD117 monoclonal antibody Indication Severe Combined Immunodeficiency Therapeutic Mechanism AMG 191 is being utilized…
Therapeutic Candidate or Device CD34+CD90+ hematopoietic stem cells (HSC) in combination with AMG 191, a humanized anti-CD117 monoclonal antibody Indication Severe Combined Immunodeficiency Therapeutic Mechanism AMG 191 is being utilized…
Therapeutic Candidate or Device Bone marrow stem cells will be transduced with a lentiviral vector to deliver a normal copy of the gamma-chain gene to treat X-linked SCID. Indication x-linked…
This video tells the story of a brave young patient, Ronnie, and his parents who decided to participate in a CIRM-funded trial run by UC San Francisco and St. Jude…
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