Gene Editing — A Cure for Transthyretin Amyloidosis?

Imagine that you had several family members with transthyretin amyloidosis. In their sixth decade of life, numbness developed, initially in their hands and feet, that progressed proximally over a period of years. This numbness came with associated muscle weakness, and then symptoms of autonomic dysfunction developed, including profound dizziness with changes in posture and alternating constipation and diarrhea that hindered their ability to engage socially. With disease progression, a syndrome of heart failure with worsening fatigue, shortness of breath, and edema developed. They initially needed an assistive device to walk, and ultimately they could not ambulate at all, losing their . . .