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Fetal gene therapy: recent advances and current challenges.
Citra N Mattar1, Mahesh Choolani, Arijit Biswas
1Yong 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.
Expert Opinion on Biological Therapy
|June 1, 2011
Summary
Fetal gene therapy (FGT) shows promise for treating severe genetic disorders before birth. While safety and ethical concerns exist, advancements in animal models suggest clinical trials may be feasible soon.
Area of Science:
- Medical Genetics
- Developmental Biology
- Gene Therapy
Background:
- Fetal gene therapy (FGT) offers potential for treating perinatally lethal monogenic diseases.
- It aims to rescue severe phenotypes and preserve fetal development.
- FGT may also induce immune tolerance for postnatal treatment.
Purpose of the Study:
- To review the role and applications of FGT.
- To assess progress in animal models simulating human development.
- To discuss challenges and factors affecting clinical translation of FGT.
Main Methods:
- Comprehensive review of existing FGT studies.
- Analysis of animal model data relevant to human development.
- Evaluation of safety, ethical considerations, and clinical translation factors.
Main Results:
- FGT has demonstrated potential in preclinical studies.
- Animal models show progress in simulating human fetal development for FGT research.
- Adverse effects on fetus and mother, and translation factors are identified.
Conclusions:
- Despite safety and ethical concerns, FGT shows significant promise.
- Evidence from non-human primate models is nearing readiness for clinical trials.
- The field is advancing towards potential human applications in the near future.
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