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Fabry disease is a rare genetic disease linked to the X chromosome.
Currently approved treatments for Fabry disease include enzyme therapy (ET) and oral molecular chaperone drug Migalastat.
In 2017, Dr.
Now, the new good news from the results of this research has been published on Nature Communications.
In this study, the researchers collected a large number of CD34+ hematopoietic stem/progenitor cells (HSPCs) from patients with Fabry disease, and then used improved lentivirus (LV)-mediated genetic means, The copy of the fully functional gene responsible for the α-Gal A enzyme is injected into these cells, and finally these modified stem cells are then transplanted back into each patient to observe the effect (NCT02800070).
Overview of the research process
Between day 6 and day 8 after infusion, the α-galA activity in the circulation was first detected in all patients, and all patients reached the reference range level (Figure a).
Figure a All patients' plasma α-gal A activity reached the reference range level; although the plasma α-gal A enzyme activity level decreased over time, it was higher than the level observed by the patient and did not return to the original baseline level ; Figure b The white blood cell α-gal A of each patient reached a supernormal level of specific activity.
The total plasma and urine Gb3 levels are variable, especially during the treatment phase.
The plasma Gb3 (a), plasma lyso-Gb3 (b), urine Gb3 (c) and urine lyso-Gb3 (d) levels of each patient.
During the entire study period, all patients had stable body weight, eGFR, proteinuria, and LVMI, except for patient No.
With these preliminary results, Health Canada approved all five patients to stop intravenous enzyme therapy.
Darren Bidulka, 52, was the first patient to receive the study on January 11, 2017.
In summary, lentivirus-mediated gene therapy may produce beneficial results for a single treatment of Fabry disease, and no serious safety issues have been found in the pilot trials.
Information source: A Canadian success story: World-first to treat Fabry disease with gene therapy
Original source: Khan, A.
