Fetal gene therapy or fetal somatic gene
therapy is emerging as a new experimental approach, in particular to prevent
irreversible perinatal disease manifestation for many inherited conditions. Fetal gene therapy 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 or protein postnatally. As the field is still at
an experimental stage, there are several important considerations regarding the
practicality and the ethics of FGT.
Know more about the
fetal gene therapy?
1. Previously
it was not very much heard as it is now in this age of modernization. Early
therapeutic gene application may also allow targeting of still expanding stem
cell populations of organ or cell systems inaccessible later in life and help
to avoid immune sensitization against the therapeutic vector system or
trans gene protein product. The progress in development of ultrasound scanning
and embryo fetoscopy over the last decade has made minimally invasive
administration of therapeutic gene transfer vectors to the fetus in utero
possible in principle.
2. Secondly
it is widely being more in trend because of the various aspects that it
carries. Given the many unknown aspects of fetal gene transfer, it is essential
to extensively investigate this new approach to gene therapy in animal models
for specific diseases, to improve on the technology of delivery and to assess
efficacy of expression as well as the possible side effects before application
to humans can be considered. The fetal gene therapy and implications is being used after it has been experimented
on animals so that it can be designed in a better way and can be used by us
without any fear.
3.
The gene
therapy for genetic disease based on the hypothesis that prenatal intervention
may avoid the development of severe manifestations of early-onset disease,
allow targeting of otherwise inaccessible tissues including expanding stem cell
populations, induce tolerance against the therapeutic transgenic protein and
thereby provide permanent somatic gene correction. This approach is
particularly relevant in relation to prenatal screening programmes for severe
genetic diseases as it could offer prevention as a third option to families
faced with the prenatal diagnosis of a genetically affected child.
4. One
of the major theoretical advantages of fetal gene therapy is the possibility of
avoiding immune reactions of the fetus against vector or trans gene product. It
is done especially since immune reactions have turned out to be one of the
major problems in adult somatic gene therapy using adeno virus vectors.
5. You
should also be aware of the fact that whether or not the human fetus will
develop an immune response against a vector and or the transgene product is
most likely to be highly dependent on the stage of fetal development at which it
is administered.
Lastly you know that the human immune system is
acquired progressively throughout the first half of pregnancy, by maturation of
the fetal immune cell repertoire in the thymus which allows self recognition
and by the development of a complete humoral and cellular immune response
against foreign antigens. And so the fetal gene therapy is being observed and
experimented so much more in recent times.
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