IN UTERO GENE THERAPY Meaning and
Definition
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In utero gene therapy is a medical procedure aimed at treating genetic disorders or diseases by introducing therapeutic genes into the fetus while it is still developing in the uterus. It involves the administration of genetic materials, such as genes or gene-editing tools, directly into the fetal tissues during pregnancy.
The primary objective of in utero gene therapy is to correct or modify genetic defects at an early stage of fetal development, potentially preventing or ameliorating the progression of inherited diseases that would otherwise manifest after birth. This procedure holds promise for a wide range of genetic conditions, including inherited metabolic disorders, immune deficiencies, and certain congenital abnormalities.
The delivery of therapeutic genes is typically achieved by injecting the genetic material into the amniotic fluid surrounding the fetus, allowing the genes to be taken up by the developing tissues, including vital organs. Alternative approaches involve direct injection into specific target tissues, such as the liver or the developing brain, depending on the nature of the disorder being targeted.
In utero gene therapy requires careful monitoring and evaluation to ensure the safety and efficacy of the procedure for both the fetus and the mother. Although still an emerging field, it holds significant potential to provide early interventions and life-long benefits for affected individuals. Research and advancements in this field are crucial in expanding our understanding of genetic diseases and developing effective strategies to improve the health outcomes of unborn babies.
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