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

被引：18

作者：

Doxzen, Kevin W. ^{[1
]}

Adair, Jennifer E. ^{[2
,3
,4
,5
]}

Fonseca Bazzo, Yris Maria ^{[6
]}

Bukini, Daima ^{[7
,8
]}

Cornetta, Kenneth ^{[9
]}

Dalal, Varsha ^{[10
]}

Guerino-Cunha, Renato Luiz ^{[11
,12
]}

Hongeng, Suradej ^{[13
]}

Jotwani, Geeta ^{[14
]}

Kityo-Mutuluuza, Cissy ^{[15
]}

Lakshmanan, Krishnamurti ^{[16
]}

Mahlangu, Johnny ^{[17
,18
]}

Makani, Julie ^{[7
,8
,19
]}

Mathews, Vikram ^{[20
]}

Ozelo, Margareth C. ^{[21
]}

Rangarajan, Savita ^{[22
,23
]}

Scholefield, Janine ^{[24
,25
]}

Silva Junior, Joao Batista ^{[6
,26
,27
]}

Mccune, Joseph M. ^{[28
]}

机构：

[1] Strateg Anal Inc, Arlington, VA 22203 USA

[2] Fred Hutchinson Canc Ctr, Translat Sci & Therapeut Div, Seattle, WA USA

[3] Univ Washington, Sch Med, Seattle, WA USA

[4] Univ Washington, Dept Lab Med, Seattle, WA USA

[5] Univ Washington, Dept Pathol, Seattle, WA USA

[6] Univ Brasilia, Fac Hlth Sci, Dept Pharmaceut Sci, Brasilia, DF, Brazil

[7] Muhimbili Univ Hlth & Allied Sci MUHAS, Dept Haematol & Blood Transfus, Sickle Cell Programme, Dar Es Salaam, Tanzania

[8] Muhimbili Univ Hlth & Allied Sci MUHAS, SickleInAfrica Clin Coordinating Ctr, Dar Es Salaam, Tanzania

[9] Indiana Univ Sch Med, Indianapolis, IN USA

[10] Minist Hlth & Family Welf, Dept Hlth Res, Govt India, New Delhi, India

[11] Ctr Paulista Oncol, Grp Oncoclin, Sao Paulo, SP, Brazil

[12] Inst Oncoclin, Rio De Janeiro, RJ, Brazil

[13] Mahidol Univ, Ramathibodi Hosp, Dept Pediat, Fac Med,Div Hematol & Oncol, Bangkok, Thailand

[14] Indian Council Med Res, New Delhi, India

[15] Joint Clin Res Ctr JCRC, Kampala, Uganda

[16] Yale Sch Med, Dept Pediat Hematol Oncol, New Haven, CT USA

[17] Univ Witwatersrand, Fac Hlth Sci, Dept Mol Med & Haematol, Johannesburg, South Africa

[18] NHLS, Johannesburg, South Africa

[19] Imperial Coll London, Ctr Haematol, Dept Immunol & Inflammat, London, England

[20] Christian Med Coll CMC, Dept Haematol, Vellore, India

[21] Univ Estadual Campinas, Sch Med Sci, Dept Internal Med, INCT Hemocentro UNICAMP, Campinas, SP, Brazil

[22] K J Somaiya Med Coll & Res Ctr, Adv Ctr Oncol Haematol & Rare Disorders ACOHRD, Mumbai, Maharashtra, India

[23] Univ Southampton, Fac Med, Southampton, Hants, England

[24] CSIR, Bioengn & Integrated Genom Grp, Next Generat Hlth Cluster, Pretoria, South Africa

[25] Univ Cape Town, Fac Hlth Sci, Dept Human Biol, Cape Town, South Africa

[26] Brazilian Hlth Regulatory Agcy ANVISA, Brasilia, DF, Brazil

[27] Univ Brasilia, Dept Pharmaceut Sci, Fac Hlth Sci, Brasilia, DF, Brazil

[28] Bill & Melinda Gates Fdn, HIV Frontiers, Global Hlth Accelerator, Seattle, WA USA

来源：

SCIENCE TRANSLATIONAL MEDICINE | 2024年 / 16卷 / 746期

基金：

比尔及梅琳达.盖茨基金会;

关键词：

CHALLENGES; BRAZIL;

D O I：

10.1126/scitranslmed.adn1902

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

摘要：

Gene therapies are designed to address the root cause of disease. As scientific understanding of disease prevention, diagnosis, and treatment improves in tandem with technological innovation, gene therapies have the potential to become safe and effective treatment options for a wide range of genetic and nongenetic diseases. However, as the medical scope of gene therapies expands, consideration must be given to those who will benefit and what proactive steps must be taken to widen development and access potential, particularly in regions carrying a high disease burden.

引用

页数：13

共 110 条

[61]

Mason C., 2016, Cell Gene Ther. Insights, V2, P141

[62] Collaborative science to advance gene therapies in resource-limited parts of the world [J].

McCune, Joseph M. ;

Stevenson, Susan C. ;

Doehle, Brian P. ;

Trenor, Cameron C., III ;

Turner, Emily H. ;

Spector, Jonathan M. .

MOLECULAR THERAPY, 2021, 29 (11) :3101-3102

[63]

MD Anderson News, 2022, Axi-cel proves effective as first-line treatment for high-risk lymphoma

[64] Evaluation of Drug Trials in High-, Middle-, and Low-Income Countries and Local Commercial Availability of Newly Approved Drugs [J].

Miller, Jennifer E. ;

Mello, Michelle M. ;

Wallach, Joshua D. ;

Gudbranson, Emily M. ;

Bohlig, Blake ;

Ross, Joseph S. ;

Gross, Cary P. ;

Bach, Peter B. .

JAMA NETWORK OPEN, 2021, 4 (05)

[65]

Ministry of Health, 2022, Anvisa approves advanced cancer therapy product registration

[66]

Ministry of Health, 2020, OrdinanceNo.1.949

[67]

Ministry of Health, 2022, Anvisa approves 3rd advanced therapy product for cancer treatment

[68]

Ministry of Health, 2022, National research with CAR-T cells to treat cancer authorized

[69]

Ministry of Health, 2022, Incorporation of medication for AME type I expands treatment of patients with the disease in the SUS

[70]

Ministry of Health, 2024, Anvisa approves first gene therapy product to treat hemophilia A in Brazil

← 2 3 4 5 6 7 8 9 10 11 →