Common pitfalls in drug target Mendelian randomization and how to avoid them.


Journal

BMC medicine
ISSN: 1741-7015
Titre abrégé: BMC Med
Pays: England
ID NLM: 101190723

Informations de publication

Date de publication:
15 Oct 2024
Historique:
received: 01 08 2024
accepted: 10 10 2024
medline: 16 10 2024
pubmed: 16 10 2024
entrez: 15 10 2024
Statut: epublish

Résumé

Drug target Mendelian randomization describes the use of genetic variants as instrumental variables for studying the effects of pharmacological agents. The paradigm can be used to inform on all aspects of drug development and has become increasingly popular over the last decade, particularly given the time- and cost-efficiency with which it can be performed even before commencing clinical studies. In this review, we describe the recent emergence of drug target Mendelian randomization, its common pitfalls, how best to address them, as well as potential future directions. Throughout, we offer advice based on our experiences on how to approach these types of studies, which we hope will be useful for both practitioners and those translating the findings from such work. Drug target Mendelian randomization is nuanced and requires a combination of biological, statistical, genetic, epidemiological, clinical, and pharmaceutical expertise to be utilized to its full potential. Unfortunately, these skillsets are relatively infrequently combined in any given study.

Sections du résumé

BACKGROUND BACKGROUND
Drug target Mendelian randomization describes the use of genetic variants as instrumental variables for studying the effects of pharmacological agents. The paradigm can be used to inform on all aspects of drug development and has become increasingly popular over the last decade, particularly given the time- and cost-efficiency with which it can be performed even before commencing clinical studies.
MAIN BODY METHODS
In this review, we describe the recent emergence of drug target Mendelian randomization, its common pitfalls, how best to address them, as well as potential future directions. Throughout, we offer advice based on our experiences on how to approach these types of studies, which we hope will be useful for both practitioners and those translating the findings from such work.
CONCLUSIONS CONCLUSIONS
Drug target Mendelian randomization is nuanced and requires a combination of biological, statistical, genetic, epidemiological, clinical, and pharmaceutical expertise to be utilized to its full potential. Unfortunately, these skillsets are relatively infrequently combined in any given study.

Identifiants

pubmed: 39407214
doi: 10.1186/s12916-024-03700-9
pii: 10.1186/s12916-024-03700-9
doi:

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

473

Informations de copyright

© 2024. The Author(s).

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Auteurs

Dipender Gill (D)

Sequoia Genetics, London, UK. dipender.gill@sequoiagenetics.com.
Department of Epidemiology and Biostatistics, School of Public Health, Imperial College London, 90 Wood Lane, London, W12 0BZ, UK. dipender.gill@sequoiagenetics.com.

Marie-Joe Dib (MJ)

Cardiovascular Division, Perelman School of Medicine, University of Pennsylvania, Philadelphia, USA.

Héléne T Cronjé (HT)

Sequoia Genetics, London, UK.
Medical Research Council Biostatistics Unit, University of Cambridge, Cambridge, UK.

Ville Karhunen (V)

Sequoia Genetics, London, UK.
Medical Research Council Biostatistics Unit, University of Cambridge, Cambridge, UK.

Benjamin Woolf (B)

Medical Research Council Biostatistics Unit, University of Cambridge, Cambridge, UK.
School of Psychological Science, University of Bristol, Bristol, UK.
Medical Research Council Integrative Epidemiology Unit, University of Bristol, Bristol, UK.

Eloi Gagnon (E)

Centre de recherche de l'Institut universitaire de cardiologie et de pneumologie de Québec, Laval University, Québec, Canada.

Iyas Daghlas (I)

Department of Neurology, University of California San Francisco, San Francisco, CA, USA.

Michael Nyberg (M)

Cardiovascular Biology, Global Drug Discovery, Novo Nordisk A/S, Maaloev, Denmark.

Donald Drakeman (D)

University of Cambridge Centre for Health Leadership & Enterprise, Judge Business School, Trumpington Street, Cambridge, UK.
Advent Venture Partners, London, UK.

Stephen Burgess (S)

Sequoia Genetics, London, UK.
Medical Research Council Biostatistics Unit, University of Cambridge, Cambridge, UK.
Cardiovascular Epidemiology Unit, Department of Public Health and Primary Care, University of Cambridge, Cambridge, UK.

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Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH