CRISPR Therapeutics and Stanford University School of Medicine researchers using CRISPR to treat sickle cell anemia, disorder affecting 100,000 people in U.S.

Briefing

CRISPR Therapeutics and Stanford University School of Medicine researchers using CRISPR to treat sickle cell anemia, disorder affecting 100,000 people in U.S.

September 6, 2017

Briefing
  • CRISPR Therapeutics – Researchers from CRIPSR Therapeutics tested CRISPR gene editing on human sickle cells in lab, and found 85% were successfully edited, able to create healthy red blood cells
  • Sickle Cell Anemia – Caused by mutation in HBB gene, protein that makes hemoglobin, resulting in abnormal blood cells that stick together and block blood vessels, resulting severe pain
  • Stanford University School of Medicine – Working on modifying HBB gene using CRISPR with clinical trials planned at end of 2018 or start of 2019
  • Disease Impact – 100,000 people is U.S. have sickle cell disease, mostly African Americans and Latinos, with people of Middle Eastern, Asian, Indian, and Mediterranean heritage also affected, shortening life span to 40-60 years

Accelerator
Biotechnology and Bioinformatics

Sector
Healthcare/Health Sciences

Organization
CRISPR Therapeutics, Stanford University

Source

Original Publication Date
August 23, 2017

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