Lentivirus-mediated gene

therapy for Fabry disease

Name:- Amina Irfan

Id:-23005254011
Program:- MS-BT
Course title:- Advances in Immunology
Journal name:- Nature Communications
Impact factor:- 17.694
 Introduction:-
• In Fabry disease, mutations of
the X-linked GLA gene lead
to accumulation of
glycosphingolipids including
globotriaosylceramide (Gb3).
• Currently approved treatment
are ERT and chaperone
therapy.
Methods:-

ist
Results :-
Conclusion:-
1. If successful, LV-mediated gene therapy may result in
beneficial outcomes for Fabry patients with a single
treatment.
2. No serious safety concerns were observed in our pilot trial.
3. All Fabry patients in this study were responsive to the LV-
mediated gene therapy at some level.
4. Gene therapy may be an effective treatment option in
patients with Fabry disease but requires more study.