First RNAi Therapy Approved by FDA
First RNAi Therapy Approved by FDA
Alnylam’s patisiran interferes with the production of a mutated protein present in people with hereditary transthyretin amyloidosis.
Alnylam’s patisiran interferes with the production of a mutated protein present in people with hereditary transthyretin amyloidosis.
hereditary transthyretin amyloidosis
Early Successes Make CRISPR-Based Medicine a Possibility
Aparna Nathan, PhD | Oct 25, 2021 | 4 min read
CRISPR-Cas9’s molecular scissors—thus far limited to the lab bench—may soon find themselves at work directly in the human body.