| Systematic Review of the Preclinical Technology Readiness of Orthopedic Gene Therapy and Outlook for Clinical Translation.
Wilkinson Piers et al. Frontiers in bioengineering and biotechnology 2021 9626315
| A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease: The Medicaid Perspective.
DeMartino Patrick et al. JAMA pediatrics 2021
| Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review.
Al-Namnam Nisreen Mohammed et al. Gene therapy 2021
| A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy.
Badawy Sherif M et al. Blood advances 2021 Jan 5(2) 570-583
| Patient and public perspectives on cell and gene therapies: a systematic review.
Aiyegbusi Olalekan Lee et al. Nature communications 2020 11(1) 6265
| Prevention methods for Treacher Collins syndrome: A systematic review.
Guo Peipei et al. International journal of pediatric otorhinolaryngology 2020 Apr 134110062
| The Potential Benefits of Deferred Payment for a Hypothetical Gene Therapy for Congestive Heart Failure: A Cost-Consequence Analysis.
Hlávka Jakub P et al. Applied health economics and health policy 2020 Feb
| CLINGEN Actionability Report for Biallelic RPE65 Mutation-Associated Retinal Dystrophy-RPE65
ClinGen Actionability Working Group
| Vascular endothelial growth factor gene transfer therapy for coronary artery disease: A systematic review and meta-analysis.
Yuan Rong et al. Cardiovascular therapeutics 2018 Oct 36(5) e12461
| Gene therapy for sickle cell disease.
Olowoyeye Abiola et al. The Cochrane database of systematic reviews 2018 Nov 11CD007652
| Gene therapy in hemophilia A: a cost-effectiveness analysis.
Machin Nicoletta et al. Blood advances 2018 Jul 2(14) 1792-1798