William Peranteau, MD, along with CHOP and Penn researchers are developing “one-and-done” new base-editing therapies for three genetic diseases of newborns.
Researchers developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.
Individuals with beta thalassemia that is treated with regular transfusions, and who are between the ages of 18 and 40 years old, may be able to participate.
The Human Pluripotent Stem Cell Core provides services for the development of novel, human cell-based experimental models for elucidating the pathophysiology of genetic disorders.
