Disease Focus: Sickle Cell Disease


Genome Editing of Autologous Hematopoietic Stem Cells to Treat Sickle Cell Disease

Therapeutic Candidate or Device Autologous blood stem cells edited to correct the sickle cell disease mutation to be given back to the patient as an autologous stem cell transplant Indication Severe sickle cell disease Therapeutic Mechanism The mechanism of the proposed therapy for sickle cell disease is that the genetically engineered autologous HSCs (pathologic S […]

Facebook Live: Stem Cells and Sickle Cell Disease

More than 80,000 Americans have sickle cell disease and despite decades of research the average life expectancy has dropped from 42 years in 1995 to 39 today. It is a disease that largely targets the African-American community and, to a lesser degree, the Hispanic community.  In recognition of September 2018 as National Sickle Awareness Month, […]

Adrienne Shapiro: Sickle Cell Disease Patient Advocate

Adrienne Shapiro is the fourth generation of mothers in her family to have children born with sickle cell disease. Ms. Shapiro was an early advocate of innovative stem cell and gene therapy work by UCLA scientist Dr. Don Kohn. This research has led to an on-going, CIRM-funded clinical trial for sickle cell disease. Ms. Shapiro […]