Applying A Public Health Lens on Gene Therapy: “Curing” Sickle Cell Disease & Beyond

Journal: Public Health Genomics

Extended Submission Deadline: January 15, 2025

Public Health Genomics publishes leading edge research and evidence-based practice at the intersection of genomic discovery and public health. Gene therapy is being lauded as the fastest growing area of innovation in genome discovery.¹ Gene therapies are treatments that edit the individual’s genome to add, remove, or otherwise modify genetic material that affects gene expression.² These therapies are experiencing a period of rapid evolvement. As of December 2022, 13 gene therapies have been approved in the U.S., 16 in the European Union and 9 in Switzerland.¹ Realizing the potential to cure conditions that until now were considered incurable, presents opportunities and challenges particularly when considering population-wide dissemination of these therapies. 

For example, SCD is the most common monogenic disease in the world. While gene therapy specific to SCD presents challenges for population dissemination globally, any of the emerging gene therapies will raise similar questions concerning: required communication (e.g., informed consent, optimistic bias about outcomes), access (e.g., cost of therapy), ethical/legal (e.g., consent), partner engagement (e.g., who and how), and generating an evidence base for dissemination of best practices (e.g., inclusion/exclusion criteria). Moreover, for sickle cell disease (SCD) in particular, but other conditions as well, opportunities and challenges will be layered with particular social, cultural, historical, and geographical contexts. ^3,4 

To that end, we are launching a call for manuscripts with the theme “Applying a Public Health Lens on Gene Therapy: “Curing” Sickle Cell Disease & Beyond” to be featured in a special collection. The goal is to highlight empirical, interventional, conceptual, ethical, and policy-based research being conducted to anticipate and address challenges of population dissemination of gene therapy to maximize public health benefit. Appropriate submissions for this article collection include but are not limited to the following questions: 

• What work is being done that exemplifies best practices for gene therapy globally (e.g., testing of gene therapy care provision that is sustainable and scalable)? 
• What actions are being taken to ensure that gene therapy is equitably accessible to all populations (e.g., public communications, public engagement, care delivery model)? 
• What governance policies, capacity-building measures, and standards of care will be needed to ensure equitable access to gene therapy? 
• How might existing public health programs (e.g., newborn screening) be capitalized on to ensure equitable access to gene therapy advances? 
• What study designs and methods will be needed to develop an evidence base to guide implementation of gene therapy to populations globally? 
• What type of institutional models might be designed to address the levels of support needed by the sickle cell population to access gene therapy? 
• How are ethical, legal, and social implications (ELSI) being addressed in the development and implementation of gene therapies for sickle cell disease.  

Please select the option “Applying A Public Health Lens on Gene Therapy: “Curing” Sickle Cell Disease & Beyond” when submitting your manuscript and mention this Call for Papers in your cover letter.

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¹Vokinger KN, Glaus CEG, Kesselheim AS. Approval and therapeutic value of gene therapies in the US and Europe. Gene Ther. 2023 Nov;30(10-11):756-760.

²Xafis V, Schaefer GO, Labude MK, Zhu Y, Holm S, Foo RSY, Lai PS, Chadwick R. Germline genome modification through novel political, ethical, and social lenses. PLoS Genet. 2021 Sep 9;17(9).

³Booth A, Bonham V, Porteus M, Ormond KE.  Treatment decision-making in sickle cell disease patients. J Community Genet. 2022 Feb;13(1):143-151.

⁴Munung NS, Nnodu OE, Moru PO, Kalu AA, Impouma B, Treadwell MJ, Wonkam A. Looking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa. Gene Ther. 2024 May;31(5-6):202-208.