|
GATA1 Gene Therapy for Diamond-Blackfan Anemia
GATA1 Gene Therapy for Diamond-Blackfan Anemia
A unified lentiviral vector based gene therapy approach, which has potential to treat DBA in all patients
Background
Diamond-Blackfan anemia (DBA) is a rare inherited disorder that that occurs when the bone marrow fails to make red blood cells, thereby leading to severe anemia.
Current therapeutic options...
Published: 3/26/2026
|
Updated: 3/9/2023
|
Inventor(s): Vijay Sankaran, Leif Ludwig, Richard Voit, David Williams
Keywords(s):
Category(s): Technology Classifications > Invention Type, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Disease Area, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Disease Area > Hematology, Technology Classifications > Invention Type > Therapeutics > Gene Therapy
|
