Fetal gene therapy

被引：9

作者：

Waddington, Simon N. ^{[1
,2
]}

Peranteau, William H. ^{[3
]}

Rahim, Ahad A. ^{[4
]}

Boyle, Ashley K. ^{[1
]}

Kurian, Manju A. ^{[5
,6
]}

Gissen, Paul ^{[7
,8
,9
]}

Chan, Jerry K. Y. ^{[10
,11
,12
]}

David, Anna L. ^{[1
]}

机构：

[1] UCL, EGA Inst Womens Hlth, London, England

[2] Wits SAMRC Antiviral Gene Therapy Res Unit, Fac Hlth Sci, Johannesburg, South Africa

[3] Childrens Hosp Philadelphia, Ctr Fetal Res, Div Gen Thorac & Fetal Surg, Philadelphia, PA USA

[4] UCL, UCL Sch Pharm, London, England

[5] UCL, GOS Inst Child Hlth, Zayed Ctr Res Rare Dis Children, Dev Neurosci, London, England

[6] Great Ormond St Hosp Sick Children, Dept Neurol, London, England

[7] UCL, Great Ormond St Inst Child Hlth, London, England

[8] Great Ormond St Hosp Children NHS Fdn Trust, London, England

[9] Great Ormond St Biomed Res Ctr, Natl Inst Hlth Res, London, England

[10] KK Womens & Childrens Hosp, Dept Reprod Med, Singapore, Singapore

[11] Duke NUS Med Sch, Acad Clin Program Obstet & Gynaecol, Singapore, Singapore

[12] Natl Univ Singapore, Yong Loo Lin Sch Med, Expt Fetal Med, Singapore, Singapore

来源：

JOURNAL OF INHERITED METABOLIC DISEASE | 2024年 / 47卷 / 01期

基金：

英国医学研究理事会;

关键词：

gene therapy; inherited metabolic disease; SEVERE COMBINED IMMUNODEFICIENCY; IN-UTERO TRANSPLANTATION; NONINVASIVE PRENATAL-DIAGNOSIS; STEM-CELL TRANSPLANTATION; SPINAL MUSCULAR-ATROPHY; HUMAN-FACTOR-IX; CLINICAL-TRIALS; BONE-MARROW; LIVER-CELLS; VECTOR;

D O I：

10.1002/jimd.12659

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Fetal gene therapy was first proposed toward the end of the 1990s when the field of gene therapy was, to quote the Gartner hype cycle, at its "peak of inflated expectations." Gene therapy was still an immature field but over the ensuing decade, it matured and is now a clinical and market reality. The trajectory of treatment for several genetic diseases is toward earlier intervention. The ability, capacity, and the will to diagnose genetic disease early-in utero-improves day by day. A confluence of clinical trials now signposts a trajectory toward fetal gene therapy. In this review, we recount the history of fetal gene therapy in the context of the broader field, discuss advances in fetal surgery and diagnosis, and explore the full ambit of preclinical gene therapy for inherited metabolic disease.

引用

页码：192 / 210

页数：19

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