title: Fetal gene therapy: recent advances and current challenges.
abstract: BACKGROUND
  Fetal gene therapy (FGT) can potentially be applied to perinatally lethal monogenic diseases for rescuing clinically severe phenotypes, increasing the probability of intact neurological and other key functions at birth, or inducing immune tolerance to a transgenic protein to facilitate readministration of the vector/protein postnatally. As the field is still at an experimental stage, there are several important considerations regarding the practicality and the ethics of FGT.
  
  
  METHODS
  Here, through a review of FGT studies, the authors discuss the role and applications of FGT, the progress made with animal models that simulate human development, possible adverse effects in the recipient fetus and the mother and factors that affect clinical translation.
  
  
  CONCLUSIONS
  Although there are valid safety and ethical concerns, the authors argue that there may soon be enough convincing evidence from non-human primate models to take the next step towards clinical trials in the near future.
authors.affiliation: Yong Loo Lin School of Medicine, National University of Singapore, Department of Obstetrics and Gynaecology, Experimental Fetal Medicine Group, NUHS Tower Block, Level 12, 1E Kent Ridge Road, 119228 Singapore.
authors.forename: Citra N
authors.initials: CN
authors.lastname: Mattar
authors.forename: Mahesh
authors.initials: M
authors.lastname: Choolani
authors.forename: Arijit
authors.initials: A
authors.lastname: Biswas
authors.forename: Simon N
authors.initials: SN
authors.lastname: Waddington
authors.forename: Jerry K Y
authors.initials: JK
authors.lastname: Chan
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D005260:Female; D005315:Fetal Diseases / Q000235:genetics* / Q000628:therapy*; D046128:Fetal Therapies / Q000379:methods* / Q000639:trends*; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends*; D005822:Genetic Vectors / Q000008:administration & dosage / Q000235:genetics; D005854:Germ Cells / Q000276:immunology; D006801:Humans; D011247:Pregnancy