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Explore Gene Therapy
Empowering the Sickle Cell Disease Community through education and awareness
Featured videos
What Are the Recent Gene Therapy Studies and Trials for Sickle Cell Disease?
- Myelodysplastic Syndrome Unrelated to Lentiviral Vector in a Patient Treated With Gene Therapy for Sickle Cell Disease
- Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease
- Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene
- Gene Therapy in a Patient With Sickle Cell Disease
- Safety and Feasibility of Hematopoietic Progenitor Stem Cell Collection by Mobilization With Plerixafor Followed by Apheresis vs Bone Marrow Harvest in Patients With Sickle Cell Disease in the Multi‐Center HGB‐206 Trial See more resources
What Are the Patient Experiences?
What Are the Centers/Hospitals Utilizing Gene Therapy for Sickle Cell Disease?
What Clinical Trials Currently Exist?
When Will Gene Therapy Be Available to More Patients?
What Should HCPs and the Medical Community Know About Gene Therapy?
- What Are the Ethical Issues Surrounding Gene Therapy?
- Gene Therapy in Clinical Trials - Cross Border Patient Transfer
- The Cost of Gene Therapy in Treating Sickle Cell Disease
- Gene Therapy a New Paradigm for Healthcare: An Interview With bluebird bio
- Professor Jennifer Doudna - CRISPR-Cas9: Genome Editing and the Future of Medicine
What Should Patients Know About Current Gene Therapy Treatments?
How Safe Is Gene Therapy?
Where Can I Find Information on Gene Therapy Clinical Studies and Trials?
- Gene Therapy Clinical Trials for Sickle Cell Disease - ClinicalTrials.Gov
- Sickle Cell Disease: Centers of Excellence and Gene Therapy
- Gene Therapy Clinical Trials - Stanford Medicine
- List of Gene Therapy Clinical Trials - Rising Tide Biology
- New and Updated Clinical Gene Therapy Trials - Gene Therapy Net
Key resources
What Are Some Examples of Approved Gene Therapies?
Who Is Working on Gene Therapy?
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Support for this section was made possible through medical education grants from Vertex and bluebird bio.
