In utero efficacy of cystic fibrosis gene therapy: difficult studies, positive or negative.
Flotte
Terence R
TR
University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA. terry.flotte@umassmed.edu
D000818:Animals; D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000235:genetics* / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D005260:Female; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D051379:Mice; D008810:Mice, Inbred C57BL; D011247:Pregnancy; D011270:Pregnancy, Animal; D015203:Reproducibility of Results; D012107:Research Design; D016896:Treatment Outcome
C505032:CFTR protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator