Genetic Disease and Treatment

General

• Gene Therapy Book HERE
• Journal HERE
• Background HERE
• Professional Organizations HERE and HERE
• Gene Therapy for Cancer HERE
• Advisory Committee HERE

Diseases

• Fanconi Anemia HERE
• Down Syndrome HERE and HERE (interesting case)
• Porphyria HERE / Background HERE
• Hemochromatosis HERE / Gene HERE
• Marfan Syndrome HERE
• Phenylketonuria HERE
• SCID HERE
• Tay-Sachs Disease HERE
• Lesch-Nyhan Syndrome HERE

Treatment Methods

• Gene Therapy - General Info HERE and HERE
• Gene Therapy for Cancer - Retroviruses and Adeno-Associated Virus

Considerations

Gene Therapy / Leukemia HERE

And what about THIS?