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The translational gap for gene therapies in low- and middle-income countries

The translational gap for gene therapies in low- and middle-income countries
The translational gap for gene therapies in low- and middle-income countries
Gene therapy is at the forefront of modern medicine. Operating at the genomic level, these sophisticated technologies are designed to address the root cause of disease. This class of medicines is made possible by advances in genetic engineering and cellular delivery technologies. As scientific understanding behind disease prevention, diagnosis, and treatment improves in tandem with technological innovation, gene therapies will hopefully become safe and effective treatment options for a wide range of genetic and non-genetic diseases. However, as the medicinal scope of gene therapies expands, consideration must be given to who will benefit and what proactive steps must be taken to widen development and access potential, particularly in areas carrying high burden diseases likely to benefit from their introduction.
1946-6234
Doxzen, Kevin W.
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Adair, Jennifer E.
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Bazzo, Yris Maria Fonseca
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Bukini, Daima
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Dalal, Varsha
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Cornetta, Kenneth
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Guerino-Cunha, Renato Luiz
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Hongeng, Suradej
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Jotwani, Geeta
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Batista, Joao
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Junior, Silva
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Kityo-Mutuluuza, Cissy
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Lakshmanan, Krishnamurti
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Mahlangu, Johnny
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Makani, Julie
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Mathews, Vikram
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Ozelo, Margareth C.
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Rangarajan, Savita
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Scholefield, Janine
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McCune, Joseph M.
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et al.
Doxzen, Kevin W.
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Adair, Jennifer E.
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Bazzo, Yris Maria Fonseca
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Bukini, Daima
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Dalal, Varsha
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Cornetta, Kenneth
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Guerino-Cunha, Renato Luiz
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Hongeng, Suradej
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Jotwani, Geeta
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Batista, Joao
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Junior, Silva
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Kityo-Mutuluuza, Cissy
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Lakshmanan, Krishnamurti
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Mahlangu, Johnny
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Makani, Julie
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Mathews, Vikram
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Ozelo, Margareth C.
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Rangarajan, Savita
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Scholefield, Janine
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McCune, Joseph M.
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Doxzen, Kevin W., Adair, Jennifer E. and Bazzo, Yris Maria Fonseca , et al. (2024) The translational gap for gene therapies in low- and middle-income countries. Science Translational Medicine. (In Press)

Record type: Article

Abstract

Gene therapy is at the forefront of modern medicine. Operating at the genomic level, these sophisticated technologies are designed to address the root cause of disease. This class of medicines is made possible by advances in genetic engineering and cellular delivery technologies. As scientific understanding behind disease prevention, diagnosis, and treatment improves in tandem with technological innovation, gene therapies will hopefully become safe and effective treatment options for a wide range of genetic and non-genetic diseases. However, as the medicinal scope of gene therapies expands, consideration must be given to who will benefit and what proactive steps must be taken to widen development and access potential, particularly in areas carrying high burden diseases likely to benefit from their introduction.

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Accepted/In Press date: 19 April 2024

Identifiers

Local EPrints ID: 489435
URI: http://eprints.soton.ac.uk/id/eprint/489435
ISSN: 1946-6234
PURE UUID: 9e39420b-194c-45b8-aece-a60d022f81b5
ORCID for Savita Rangarajan: ORCID iD orcid.org/0000-0001-7367-133X

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Date deposited: 24 Apr 2024 16:34
Last modified: 25 Apr 2024 01:57

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Contributors

Author: Kevin W. Doxzen
Author: Jennifer E. Adair
Author: Yris Maria Fonseca Bazzo
Author: Daima Bukini
Author: Varsha Dalal
Author: Kenneth Cornetta
Author: Renato Luiz Guerino-Cunha
Author: Suradej Hongeng
Author: Geeta Jotwani
Author: Joao Batista
Author: Silva Junior
Author: Cissy Kityo-Mutuluuza
Author: Krishnamurti Lakshmanan
Author: Johnny Mahlangu
Author: Julie Makani
Author: Vikram Mathews
Author: Margareth C. Ozelo
Author: Janine Scholefield
Author: Joseph M. McCune
Corporate Author: et al.

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