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