Last Posted: Feb 26, 2021
- Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review.
Al-Namnam Nisreen Mohammed et al. Gene therapy 2021
- Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision.
Huygens Simone A et al. PharmacoEconomics 2021 Feb
- Gene therapy trials for sickle cell disease halted after two patients develop cancer
J Kaiser, Science, February 16, 2021
- Safety of Intravitreal Gene Therapy for Treatment of Subjects with Leber Hereditary Optic Neuropathy due to Mutations in the Mitochondrial ND4 Gene: The REVEAL Study.
Vignal-Clermont Catherine et al. BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy 2021 Feb
- Gene therapy for inherited metabolic diseases.
Yilmaz Berna Seker et al. Journal of mother and child 2020 Nov 24(2) 53-64
- Spinal Muscular Atrophy: Mutations, Testing, and Clinical Relevance.
Keinath Melissa C et al. The application of clinical genetics 2021 1411-25
- Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy
V Himic et al, EJHG, February 9, 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
- Suppression-Replacement KCNQ1 Gene Therapy for Type 1 Long QT Syndrome.
Dotzler Steven M et al. Circulation 2021 Jan
- Analysis of Pathogenic Variants Correctable With CRISPR Base Editing Among Patients With Recessive Inherited Retinal Degeneration.
Fry Lewis E et al. JAMA ophthalmology 2021 Jan
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Disclaimer: Articles listed in the Public Health Genomics and Precision Health Knowledge Base are selected by the CDC Office of Public Health Genomics to provide current awareness of the literature and news. Inclusion in the update does not necessarily represent the views of the Centers for Disease Control and Prevention nor does it imply endorsement of the article's methods or findings. CDC and DHHS assume no responsibility for the factual accuracy of the items presented. The selection, omission, or content of items does not imply any endorsement or other position taken by CDC or DHHS. Opinion, findings and conclusions expressed by the original authors of items included in the update, or persons quoted therein, are strictly their own and are in no way meant to represent the opinion or views of CDC or DHHS. References to publications, news sources, and non-CDC Websites are provided solely for informational purposes and do not imply endorsement by CDC or DHHS.