The Patients' Stories

Successful Case Stories, Published in Scientific Journals

First Successful Gene Therapy for Infant with Severe Combined Immune Deficiency (SCID)

January 24, 2023
The first successful gene therapy of a baby with severe combined immune deficiency (SCID)

Severe Combined Immunodeficiency (SCID), stemming from Adenosine deaminase (ADA) enzyme deficiency, proves fatal due to inevitable infections, earning it the moniker “bubble baby” for the stringent isolation required to safeguard against contagion.

The sole potential cure lies in successful gene therapy, wherein the ADA gene is introduced into multipotent stem cells responsible for immune system development. Despite prior failures in such endeavors, a glimmer of hope emerged in 2002 when Prof. Slavin encountered a newborn afflicted with ADA-deficient SCID. Tragically, a sibling had succumbed to recurrent infections prior to diagnosis, spurring urgent intervention.

With no suitable donor available, Prof. Slavin embarked on a groundbreaking gene therapy endeavor. Diverging from conventional vector-based approaches, he opted for the transplantation of the patient’s own ADA-transduced cord blood and bone marrow stem cells.

The newborn, Tasnin, became the beacon of success in 2002, marking the inaugural triumph of gene therapy for ADA-deficient SCID. A meticulous protocol, initiated with harmless non-myeloablative conditioning, paved the way for Tasnin’s immune system revitalization. Rapid engraftment and differentiation of genetically engineered stem cells ensued, propelling her towards seamless recovery sans additional interventions.

Tasnin’s journey epitomizes Prof. Slavin’s pioneering method, ensuring consistent engraftment and multi-lineage differentiation of genetically modified stem cells. The accompanying photos capture Tasnin, flanked by her sister Salsabil and mother, embodying hope and resilience four years post-treatment.

In Tasnin’s triumphant tale lies a testament to the transformative potential of gene therapy in combatting SCID. Alive and thriving since 2002, she stands as a living testament to the power of innovation and perseverance in the face of adversity.

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