In utero gene therapy: current challenges and perspectives.
Over the past few years, considerable progress in prenatal diagnosis and surgery combined with improvements in vector design vindicate a reappraisal of the feasibility of in utero gene therapy for serious monogenetic diseases. As adult gene therapy gathers pace, several apparent obstacles to its application as a treatment may be overcome by pre- or early postnatal treatment. This review will examine the concepts and practice of prenatal vector administration. We aim to highlight the advantages of early therapeutic intervention focusing on diseases that could benefit greatly from a prenatal gene therapy approach. We will pay special attention to the strategies and vectors that are most likely to be used for this application and will speculate on their expected developments for the near future.
Waddington
Simon N
SN
Gene Therapy Research Group, Sir Alexander Fleming Building, Imperial College, South Kensington, London SW7 2AZ, UK.
Kramer
M Gabriela
MG
Hernandez-Alcoceba
Ruben
R
Buckley
Suzanne M K
SM
Themis
Michael
M
Coutelle
Charles
C
Prieto
Jesus
J
D000818:Animals; D005260:Female; D005333:Fetus / Q000378:metabolism*; D030342:Genetic Diseases, Inborn / Q000235:genetics / Q000628:therapy; D015316:Genetic Therapy / Q000009:adverse effects / Q000941:ethics / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage / Q000235:genetics; D006801:Humans; D011247:Pregnancy; D014599:Uterus / Q000378:metabolism*