Last Posted: May 06, 2021
- Optimizing language for effective communication of gene therapy concepts with hemophilia patients: a qualitative study.
Hart Daniel P et al. Orphanet journal of rare diseases 2021 16(1) 189
- Current Status of Gene Therapy Research in Polyglutamine Spinocerebellar Ataxias.
Afonso-Reis Ricardo et al. International journal of molecular sciences 2021 22(8)
- Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies.
Garrison Louis P et al. Journal of managed care & specialty pharmacy 2021 27(5) 674-681
- Hematopoietic stem cell transplantation for people with β-thalassaemia.
Sharma Akshay et al. The Cochrane database of systematic reviews 2021 4CD008708
- Recent Advancements in the Diagnosis and Treatment of Leukodystrophies.
Bradbury Allison M et al. Seminars in pediatric neurology 2021 37100876
- Spinal Muscular Atrophy.
Nicolau Stefan et al. Seminars in pediatric neurology 2021 37100878
- Gene therapy needs a long-term approach
Editorial, Nature Medicine, April 16, 2021
- CNGB1-related rod-cone dystrophy: a mutation review and update.
Nassisi Marco et al. Human mutation 2021
- [Progress in Gene Therapy of Sickle Cell Disease Based on Hemoglobin F--Review].
Liang Hao et al. Zhongguo shi yan xue ye xue za zhi 2021 29(2) 643-647
- 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
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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.