A new review accepted for publication! Hermans et al. 2023

Congratulations to Pr. Cédric Hermans and colleagues for the publication by the journal Therapeutic Advances in Hematology of their review on gene therapy in hemophilia. We are very glad that we have been able to participate to the success of this review!

Hemophilia is an genetic disorder in which the blood cannot clot. There are two types of hemophilia: hemophilia A, characterized by a deficiency of coagulation factor VIII, and hemophilia B, which is related to a deficiency of coagulation factor IX. These two types of hemophilia respectively affect one boy in 5,000 and one boy in 25,000. This disease is transmitted by the X chromosome, so female individuals are rarely affected.

Until now, treatments have focused on replacing missing coagulation factors. More recently, gene therapy has proven to be a new and promising solution in the treatment of patients with hemophilia. It allows patients to produce the missing clotting factor by providing them with a functional version of the mutated gene.

Although this approach represents a major advance, several issues need to be resolved before this therapy can be implemented in routine clinical practice. This review discusses recent advances in the field and the hurdles that must be overcome to allow as many patients as possible to benefit from this therapeutic solution.

The article is already available on the journal's website: Hermans C, Gruel Y, Frenzel L, Krumb E. How to translate and implement the current science of gene therapy into haemophilia care? Ther Adv Hematol. 2023 Jan 12;14:20406207221145627. doi: 10.1177/20406207221145627.