Gene Therapy for Monogenic Inherited Disorders

CONCLUSION: The first gene treatments have now been approved and bring hope of long-term therapeutic benefit after a single administration. The numbers of patients who come into question for specific therapies are often low, so that many different aspects- generation of evidence on efficacy and safety, determining indications, performance of the treatment, pricing-bring new challenges.PMID:33637169 | DOI:10.3238/arztebl.2020.0878
Source: Deutsches Arzteblatt International - Category: General Medicine Authors: Source Type: research