Last Posted: Sep 19, 2019
- Future Preventive Gene Therapy of Polygenic Diseases from a Population Genetics Perspective
RT Oliynyk, BioRXIV, September 18, 2019
- Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism.
Ginocchio Virginia Maria et al. Human gene therapy 2019 Sep
- Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia.
Río Paula et al. Nature medicine 2019 Sep 25(9) 1396-1401
- Update on gastric cancer treatments and gene therapies.
Biagioni Alessio et al. Cancer metastasis reviews 2019 Sep
- CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia.
Xu Lei et al. The New England journal of medicine 2019 Sep
- First gene therapy for β-thalassemia approved
C Harrison, Nature Biotechnology, September 9, 2019
- Gene Therapy for Choroideremia-Progress and Remaining Questions.
Duncan Jacque L et al. JAMA ophthalmology 2019 Aug
- Important Considerations in Modeling the Cost-Effectiveness for the First Food and Drug Administration-Approved Gene Therapy and Implications for Future One-Time Therapies.
Buessing Marric et al. Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research 2019 Aug 22(8) 970-971
- Long QT Syndrome: Genetics and Future Perspective.
Wallace Eimear et al. Pediatric cardiology 2019 Aug
- Mom says she'll fight for world's most expensive drug for daughter, costs $2 million
AJC, August 22, 2019
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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.
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