Gene Therapy

Recent News

What Happens When CRISPR Backfires?

Gene editing technology may not be as safe as we thought. Human trials only taken place in China so far, but no results published. FDA placed a clinical hold on first US trial. CRISPR-Cas9 works by cutting the DNA at a designated spot. New studies suggest CRISPR can damage or kill cells. In 20% of cells CRISPR results in much larger deletions than expected. Researchers had only looked for mutations in immediate vicinity of the cut. New techniques - REPAIR, Cas13 and ABE7 may overcome these problems.

Drug approved as alternative method to CRISPR

FDA approve new family of drugs able to cure inherited diseases. Alnylam has created patisiran to treat genetic nerve damage. Based on RNA interference (RNAi) which can silence genes. RNAi can potentially cure any disease caused by “bad” proteins. These diseases include stroke, Alzheimer’s, and high cholesterol. Targets root cause of condition rather than just its symptoms.

Caution Regarding CRISPR Gene Therapies

CRISPR can cause more unintended damage than thought. Enzyme cuts a cell’s DNA and relies on the cell to reconnect. Can cause large deletions or rearrangements of DNA around the target site. Fears this kind of damage to DNA could lead to cancer. Has gone unnoticed as looking for small mutations. "Changes in the DNA have been seriously underestimated”