BMJ Evidence-Based Medicine
August 2021 – Volume 26 – 4
An evidence map of randomised controlled trials evaluating genetic therapies (10 November, 2020)
Genetic therapies replace or inactivate disease-causing genes or introduce new or modified genes. These therapies have the potential to cure in a single application rather than treating symptoms through repeated administrations. This evidence map provides a broad overview of the genetic therapies that have been evaluated in randomised controlled trials (RCTs) for efficacy and safety.
Eric A. Apaydin, Andrea S. Richardson, Sangita Baxi, Jerry Vockley, Olamigoke Akinniranye, Rachel Ross, Jody Larkin, Aneesa Motala, Gulrez Azhar, Susanne Hempel