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Fetal gene therapy.
Waddington, Simon N; Peranteau, William H; Rahim, Ahad A; Boyle, Ashley K; Kurian, Manju A; Gissen, Paul; Chan, Jerry K Y; David, Anna L.
Afiliação
  • Waddington SN; EGA Institute for Women's Health, University College London, London, UK.
  • Peranteau WH; Faculty of Health Sciences, Wits/SAMRC Antiviral Gene Therapy Research Unit, Johannesburg, South Africa.
  • Rahim AA; The Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
  • Boyle AK; UCL School of Pharmacy, University College London, London, UK.
  • Kurian MA; EGA Institute for Women's Health, University College London, London, UK.
  • Gissen P; Developmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, GOS-Institute of Child Health, University College London, London, UK.
  • Chan JKY; Department of Neurology, Great Ormond Street Hospital for Children, London, UK.
  • David AL; Great Ormond Street Institute of Child Health, University College London, London, UK.
J Inherit Metab Dis ; 47(1): 192-210, 2024 Jan.
Article em En | MEDLINE | ID: mdl-37470194
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.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Terapias Fetais Limite: Female / Humans / Pregnancy Idioma: En Ano de publicação: 2024 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Terapias Fetais Limite: Female / Humans / Pregnancy Idioma: En Ano de publicação: 2024 Tipo de documento: Article