Cell and Gene Therapy Innovations Patient Stories

Building on our proven track record in cell and gene therapy, CHOP is committed to discovering the next life-changing treatments for the most serious diseases.

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Patient story

Experimental Gene Therapy for Hemophilia B: Bill’s Story

Bill’s hemophilia caused several serious health crises. After nearly 60 years of continual treatments, a gene therapy transformed his life.

Patient story

Experimental Gene Therapy for Hemophilia B: Jay’s Story

For 50-plus years, Jay dealt with the frequent treatments needed for his hemophilia. A gene therapy put an end to the treatments and to the worry the disease caused him.

Patient story

Experimental Gene Therapy for Beta Thalassemia: Aliya’s Story

For the first 30 years of her life, Aliya needed monthly blood transfusions for her inherited blood disorder. A gene therapy changed everything.

Patient story

Gene Therapy Treatment for Spinal Muscular Atrophy: Céline’s Story

Céline’s symptoms dramatically improved after she received a new gene therapy treatment for Type 1 spinal muscular atrophy (SMA) at CHOP.

Patient story

Gene Therapy for Inherited Blindness: Hannah’s Leber Congenital Amaurosis Story

Diagnosed with Leber congenital amaurosis as a baby, Hannah was on a path to going blind until gene therapy at Children’s Hospital restored her sight.

Patient story

CAR T-Cell Therapy for Relapsed Leukemia: Paulina’s Story

Frantic to save Paulina’s life, her family brought her to the Cancer Center at CHOP where an experimental trial of CAR T-cell therapy cured her cancer.