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Last Posted: Sep 29, 2022
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Sickle Cell Disease and Gene Therapy - Patient and Physician Perspectives.
DeBaun Michael et al. The New England journal of medicine 2022 9 (13) e28

In this short documentary video, patients and physicians partner both to highlight the experience of living with sickle cell disease and to discuss the pathophysiology of the disease and new treatment strategies, including gene therapy. Patients share their own stories of interactions with the health care system and explore the challenging topics of racial disparity and health equity. Physicians express a cautious optimism as they review the risks and benefits of gene therapy.

Vital Signs: Use of Recommended Health Care Measures to Prevent Selected Complications of Sickle Cell Anemia in Children and Adolescents — Selected U.S. States, 2019
LA Schieve et al, CDC, MMWR, September 20, 2022

Sickle cell anemia (SCA), which primarily affects Black or African American persons, is associated with severe complications and reduced life expectancy. Among children and adolescents with SCA, transcranial Doppler (TCD) ultrasound screening identifies elevated risk for stroke, and hydroxyurea therapy can reduce the occurrence of several life-threatening complications. During 2019, fewer than one half of Medicaid enrollees aged 2–16 years with SCA had a TCD screen. Fewer than one half of children aged 2–9 years used hydroxyurea and approximately one half of those aged 10–16 years used hydroxyurea.

Treatment of Genetic Diseases With CRISPR Genome Editing
MJ Kan et al, JAMA, September 13, 2022

Ongoing clinical trials use CRISPR for somatic cell genome editing to treat hereditary diseases or cancer. In these studies, cells are either removed and edited in tissue culture and then readministered to the patient (ex vivo), or genome editors are packaged within viral vectors or lipid nanoparticles and given intravenously to home to specific tissues (in vivo). Ex vivo therapies have focused on blood disorders, including at least 6 trials aiming to cure sickle cell disease (SCD) or transfusion-dependent ß-thalassemia (TDT).

National Sickle Cell Awareness Month
HHS Minority Health, September 2022 Brand

September is National Sickle Cell Awareness Month, a time to recognize the perseverance of patients living with sickle cell disease (SCD) and to recommit ourselves to improving the quality of life and health outcomes for all individuals living with SCD. Learn how federal agencies are working to put an end to SCD.


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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