[HTML][HTML] A review of existing strategies for designing long-acting parenteral formulations: Focus on underlying mechanisms, and future perspectives

Y Shi, A Lu, X Wang, Z Belhadj, J Wang… - … Pharmaceutica Sinica B, 2021 - Elsevier
The need for long-term treatments of chronic diseases has motivated the widespread
development of long-acting parenteral formulations (LAPFs) with the aim of improving drug …

[HTML][HTML] A review of the rationale for gene therapy for hemophilia A with inhibitors: one-shot tolerance and treatment?

LA Valentino, MC Ozelo, RW Herzog, NS Key… - Journal of Thrombosis …, 2023 - Elsevier
The therapeutic landscape for people living with hemophilia A (PwHA) has changed
dramatically in recent years, but many clinical challenges remain, including the development …

Immune tolerance against infused FVIII in hemophilia A is mediated by PD-L1+ Tregs

J Becker-Gotot, M Meissner, V Kotov… - The Journal of …, 2022 - Am Soc Clin Investig
A major complication of hemophilia A therapy is the development of alloantibodies
(inhibitors) that neutralize intravenously administered coagulation factor VIII (FVIII). Immune …

Factor VIII trafficking to CD4+ T cells shapes its immunogenicity and requires several types of antigen-presenting cells

R Kaczmarek, AR Piñeros, PE Patterson… - Blood, The Journal …, 2023 - ashpublications.org
Despite> 80 years of clinical experience with coagulation factor VIII (FVIII) inhibitors,
surprisingly little is known about the in vivo mechanism of this most serious complication of …

B cell focused transient immune suppression protocol for efficient AAV readministration to the liver

J Rana, RW Herzog, M Muñoz-Melero… - … Therapy Methods & …, 2024 - cell.com
Adeno-associated virus (AAV) vectors are used for correcting multiple genetic disorders.
Although the goal is to achieve lifelong correction with a single vector administration, the …

On the road to eliminating long‐lived plasma cells—“are we there yet?”

C Markmann, VG Bhoj - Immunological Reviews, 2021 - Wiley Online Library
Central to protective humoral immunity is the activation of B cells and their terminal
differentiation into antibody‐secreting plasma cells. Long‐lived plasma cells (LLPC) may …

Increased B Cell‐Activating Factor Expression Is Associated with Postoperative Recurrence of Chronic Rhinosinusitis with Nasal Polyps

F Zhang, Z Xu, X He, Y Sun, C Zhao… - Mediators of …, 2022 - Wiley Online Library
Background. Chronic rhinosinusitis with nasal polyps (CRSwNP) is a common upper airway
inflammatory disease with a high postoperative recurrence rate. This study is aimed at …

Hemophilia a patients with inhibitors: Mechanistic insights and novel therapeutic implications

L Luo, Q Zheng, Z Chen, M Huang, L Fu, J Hu… - Frontiers in …, 2022 - frontiersin.org
The development of coagulation factor VIII (FVIII) inhibitory antibodies is a serious
complication in hemophilia A (HA) patients after FVIII replacement therapy. Inhibitors render …

Coagulation factor VIII: biological basis of emerging hemophilia A therapies

BJ Samelson-Jones, BS Doshi, LA George - Blood, 2024 - ashpublications.org
Coagulation factor VIII (FVIII) is essential for hemostasis. After activation, it combines with
activated FIX (FIXa) on anionic membranes to form the intrinsic Xase enzyme complex …

Abnormal frequency of the memory B cell subsets and plasmablasts in patients with congenital severe hemophilia A: correlation with “Inhibitor” formation

OR Zekavat, Y Movahednezhad, A Shahsavani… - Blood Research, 2024 - Springer
Background Development of antibodies against infused Factor VIII (FVIII) or" inhibitors"
represents a major challenge following FVIII replacement therapy in patients with …