Therapeutic potential of siRNA PMP22-SQ nanoparticles for Charcot-Marie-Tooth 1A neuropathy in rodents and non-human primates

Authors
Suzan Boutary, Guy Khalaf, Yosef Landesman, Mevidette el Madani, Didier Desmaële, Françoise Piguet, Rafael Alonso, Elena-Gaia Banchi, David Adams, Charbel Massaad, Liliane Massaad-Massade


Lab

Journal
International Journal of Pharmaceutics

Abstract
Small interfering RNA (siRNA) has shown promising results for the treatment of Charcot-Marie-Tooth disease 1A (CMT1A) caused by overexpression of peri…

Keywords/Topics
Small interfering RNA;Nanoparticles;Squalene;Charcot Marie Tooth 1A;Non-Human Primates;Biodistribution;Pharmacokinetics;Safety Profile

BIOSEB Instruments Used:
Grip strength test (BIO-GS4)

Source :

https://www.sciencedirect.com/science/article/pii/S0378517325000705

Related products

Publication request

Thank you for your interest in our product range and your request for this publication, which will be sent to you if the research team and the journal allow it. Our commercial team will contact you as soon as possible.