Gene therapy access: Global challenges, opportunities, and views from Brazil, South Africa, and India

被引：36

作者：

Cornetta, Kenneth ^{[1
]}

Bonamino, Martin ^{[2
,3
]}

Mahlangu, Johnny ^{[4
]}

Mingozzi, Federico ^{[5
]}

Rangarajan, Savita ^{[6
,7
]}

Rao, Jayandharan ^{[8
]}

机构：

[1] Indiana Univ Sch Med, Dept Med & Mol Genet, Indianapolis, IN 46202 USA

[2] Natl Canc Inst INCA, Res Coordinat, Mol Carcinogenesis Program, Rio De Janeiro, Brazil

[3] Oswaldo Cruz Fdn FIOCRUZ, Vice Presidency Res & Biol Collect VPPCB, Rio De Janeiro, Brazil

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

[5] Spark Therapeut, Philadelphia, PA 19104 USA

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

[7] KJ Somaiya Super Special Hosp & Res Ctr, Mumbai, Maharashtra, India

[8] Indian Inst Technol Kanpur, Dept Biol Sci & Bioengn, Kapur, UP, India

来源：

MOLECULAR THERAPY | 2022年 / 30卷 / 06期

关键词：

BREAST-CANCER; CELL; HEMOPHILIA; DIAGNOSIS; COUNTRIES;

D O I：

10.1016/j.ymthe.2022.04.002

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Gene and cell therapies for a variety of life-limiting illnesses are under investigation, and a small number of commercial products have successfully obtained regulatory approval. The cost of treatment is high, and clinical studies evaluating safety and efficacy are performed predominately in high-income countries. We reviewed the current status of gene and cell therapies in low- and middle-income countries and highlighted the need and current barriers to access. The state of product development in Brazil, South Africa, and India is discussed, including lessons learned from American Society of Gene and Cell Therapy (ASGCT)-sponsored virtual symposia in each of these countries.

引用

页码：2122 / 2129

页数：8

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