Evidence map›Paper›PMID 38012299›Full record

ReviewGene therapy2024

Looking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa.

Nchangwi Syntia Munung, Obiageli E Nnodu, Patrick Ohiani Moru, Akpaka A Kalu, Benido Impouma, Marsha J Treadwell, Ambroise Wonkam

Open access · hybridAbstract readReview
In one paragraph

Review in Gene therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed, 2 pooled it
5.5field-weighted citation impact, top 4% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

21 citing papers in PubMed, 2 syntheses or guidelines pooled it, 19 citations in OpenAlex.

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  18. Genome editing strategies for targeted correction of β-globin mutation in sickle cell disease: From bench to bedside.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors at 5 institutions in 4 countries.

Nchangwi Syntia MunungDivision of Human Genetics, Faculty of Health Sciences, University of Cape Town, Cape Town, South Africa. munung.nchangwi@uct.ac.za.ORCID 0000-0003-1498-3602
Obiageli E NnoduCentre of Excellence for Sickle Cell Disease Research and Training (CESRTA), University of Abuja, Abuja, Nigeria.ORCID 0000-0002-4801-3156
Patrick Ohiani MoruCentre of Excellence for Sickle Cell Disease Research and Training (CESRTA), University of Abuja, Abuja, Nigeria.ORCID 0009-0001-1440-5143
Akpaka A KaluWorld Health Organization Regional Office for Africa, Brazzaville, Republic of Congo.
Benido ImpoumaWorld Health Organization Regional Office for Africa, Brazzaville, Republic of Congo.
Marsha J TreadwellDepartment of Pediatrics, Division of Hematology, University of California San Francisco, Oakland, CA, USA.
Ambroise WonkamDivision of Human Genetics, Faculty of Health Sciences, University of Cape Town, Cape Town, South Africa. awonkam1@jhmi.edu.
University of Abuja · NGWorld Health Organization Regional Office for Africa · CGJohns Hopkins University · USUniversity of California, San Francisco · USUniversity of Cape Town · ZA

Funding

Sickle Africa Data Coordinating CenterU24HL135600 · NHLBI · UNIVERSITY OF CAPE TOWN · PI MULDER, NICOLA, WONKAM, AMBROISE · 2017 to 2025
$8.6M
Public Understanding of Big data in Genomics Medicine in Africa (PUBGEM-Africa)U01MH127692 · NIMH · UNIVERSITY OF CAPE TOWN · PI MUNUNG, NCHANGWI SYNTIA, WONKAM, AMBROISE · 2021 to 2025
$1.9M
Sickle Pan African Research Consortium NigEria (SPARC-NEt)U01HL156942 · NHLBI · UNIVERSITY OF ABUJA · PI NNODU, OBIAGELI EUNICE · 2021 to 2025
$1.3M
NHLBI NIH HHS U01 HL156942NHLBI NIH HHS U24 HL135600NIMH NIH HHS U01 MH127692World Health Organization 001
6 · The paper itself

Abstract

Somatic gene therapy will be one of the most exciting practices of genetic medicine in Africa and is primed to offer a "new life" for persons living with sickle cell disease (SCD). Recently, successful gene therapy trials for SCD in the USA have sparked a ray of hope within the SCD community in Africa. However, the high cost, estimated to exceed 1.5 million USD, continues to be a major concern for many stakeholders. While affordability is a key global health equity consideration, it is equally important to reflect on other ethical, legal and social issues (ELSIs) that may impact the responsible implementation of gene therapy for SCD in Africa. These include informed consent comprehension, risk of therapeutic misestimation and optimistic bias; priorities for SCD therapy trials; dearth of ethical and regulatory oversight for gene therapy in many African countries; identifying a favourable risk-benefit ratio; criteria for the selection of trial participants; decisional conflict in consent; standards of care; bounded justice; and genetic tourism. Given these ELSIs, we suggest that researchers, pharma, funders, global health agencies, ethics committees, science councils and SCD patient support/advocacy groups should work together to co-develop: (1) patient-centric governance for gene therapy in Africa, (2) public engagement and education materials, and (3) decision making toolkits for trial participants. It is also critical to establish harmonised ethical and regulatory frameworks for gene therapy in Africa, and for global health agencies to accelerate access to basic care for SCD in Africa, while simultaneously strengthening capacity for gene therapy.

Indexed as

Anemia, Sickle CellGenetic TherapyAfricaHumansInformed Consent

Identifiers

PMID38012299
PMCPMC11090833
OpenAlexW4389042087

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.