Mechanism of fetal gene therapy
•In general, is addition of a new gene to the cell to
compensate for a mutated gene by viral vectors (AAV).
•its mechanism is a working gene is transferred
into the cell by viral vectors to start produce a
normal protein
There is two ways to give gene therapy
❖In vivo:
Injection of gene inside the body
❖Ex vivo:
Injection of gene outside the body
(in laboratory)
What can be accomplished by gene
therapy ?
Scientists are looking to treat many genetic diseases
through fetal gene therapy such as :-
5-Gaucher’s disorder
2-SCID
3-Dawn’s syndrome
4-Haemophilia
1-XLHED
❖In2018studypublishedinTheNewEnglandJournalof
Medicineresearchersusedfetalgenetherapytoprevent
XIHEDaninheriteddisorderinthreebabies.
❖The babies were around 2 years old when paper was
published and seemed to be okay.
Risks of Gene Therapy
1-the most common problem is a lack of
effectiveness.
2-(Unwanted Immune System Reaction )
3-(Wrong Target Cell)
4-(Infection Caused by Viral Vector)
5-(possibility of unintended genetic changes)
6-(Possible Tumor)