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.
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
istock-845814134-800x560
Early Successes Make CRISPR-Based Medicine a Possibility
Aparna Nathan | Oct 25, 2021
CRISPR-Cas9’s molecular scissors—thus far limited to the lab bench—may soon find themselves at work directly in the human body.