Last Posted: Jun 14, 2019
- Advances in the Treatment of Sickle Cell Disease.
Kapoor Sargam et al. Mayo Clinic proceedings 2018 93(12) 1810-1824
- Wilson disease-treatment perspectives.
Litwin Tomasz et al. Annals of translational medicine 2019 Apr 7(Suppl 2) S68
- Assessing the Joint Value of Genomic-Based Diagnostic Tests and Gene Therapies.
Gavan Sean P et al. Journal of personalized medicine 2019 May 9(2)
- How to discuss gene therapy for haemophilia? A patient and physician perspective.
Miesbach Wolfgang et al. Haemophilia : the official journal of the World Federation of Hemophilia 2019 May
- New molecular therapies for the treatment of hearing loss.
Ma Yutian et al. Pharmacology & therapeutics 2019 May
- At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever
R Stein, NPR, May 24, 2019
- Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients.
Paquin Ryan S et al. Orphanet journal of rare diseases 2019 May 14(1) 102
- Getting serious about the challenge of regulating germline gene therapy.
Caplan Arthur et al. PLoS biology 2019 Apr 17(4) e3000223
- Inherited Platelet Disorders: A Modern Approach to Evaluation and Treatment.
Lambert Michele P et al. Hematology/oncology clinics of North America 2019 Jun 33(3) 471-487
- A Genetics Learning Program for Nurses Caring for Children Treated With Ex Vivo Autologous Gene Therapy.
Williams Lucinda et al. Journal of continuing education in nursing 2019 May 50(5) 218-227
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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.
- Page last reviewed:Mar 1, 2018
- Page last updated:Jun 19, 2019
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